ALAMEDA, Calif.--(BUSINESS WIRE)--AgeX Therapeutics, Inc. (AgeX; NYSE American: AGE), a biotechnology company developing therapeutics for human aging and regeneration, reported its financial and operating results for the fourth quarter and year-end results for 2019.

Additional Recent Highlights

Balance Sheet Information

Cash, and cash equivalents, and restricted cash totaled $2.5 million as of December 31, 2019, as compared with $6.7 million as of December 31, 2018.

AgeX is in need of additional capital to finance its operations. On March 30, 2020, AgeX entered into a Secured Convertible Facility Agreement (the Loan Facility) with Juvenescence Limited pursuant to which AgeX may borrow funds from time to time. Loans from Juvenescence in excess of an initial $500,000 advance will be subject to Juvenescences discretion. If AgeX makes a second $500,000 draw under the Loan Facility, it will be required to implement a cost reduction plan that will entail significant reductions in staffing and research and development activities, and if a third advance of funds is approved by Juvenescence, AgeX and certain of its subsidiaries will be required to enter into a Security and Pledge Agreement pursuant to which they will pledge substantially all of their assets to collateralize all loans drawn under the Loan Facility. AgeX will issue stock purchase warrants to Juvenescence based on the amount of loans Juvenescence makes, and will issue 28,500 shares of AgeX common stock to Juvenescence if Juvenescence lends AgeX $3 million in the aggregate. Juvenescence will also have the right to convert outstanding loan balances into shares of AgeX common stock at market prices. More information about the Loan Facility can be found in AgeXs Annual Report on Form 10-K filed with the Securities and Exchange Commission.

As required under Accounting Standards Update 2014-15, Presentation of Financial Statements-Going Concern (ASC 205-40), AgeX evaluates whether conditions and/or events raise substantial doubt about its ability to meet its future financial obligations as they become due within one year after the date its financial statements are issued. Based on AgeXs most recent projected cash flows, and considering that loans from Juvenescence in excess of an initial $500,000 advance under the Loan Facility will be subject to Juvenescences discretion, AgeX believes that its cash and cash equivalents and a $500,000 loan under the Loan Facility would not be sufficient to satisfy its anticipated operating and other funding requirements for the twelve months following the filing of the Form 10-K. These factors raise substantial doubt regarding the ability of AgeX to continue as a going concern, and the report of AgeXs independent registered public accountants accompanying the audited financial statements in AgeXs Annual Report on Form 10-K contains a qualification to such effect.

Fourth Quarter and Annual 2019 Operating Results

Revenues: Total Revenues for the fourth quarter of 2019 were $0.5 million as compared to $0.3 million in the comparable quarter in 2018. Total revenues for the year ended December 31, 2019 were $1.73 million, as compared with $1.4 million in the same period in 2018, representing an increase of approximately 24%. AgeX revenue is primarily generated from subscription and advertising revenues from the GeneCards online database through its subsidiary LifeMap Sciences, Inc. Revenues for the year ended December 31, 2019 also included approximately $180,000 of allowable expenses under its research grant from the NIH as compared with $20,000 in the same period in 2018.

Operating expenses: Operating expenses for the three months ended December 31, 2019, were $3.2 million, as reported, which was comprised of $2.7 million for AgeX and $0.5 million for LifeMap Sciences, and were $2.5 million, as adjusted, comprised of $2.1 million for AgeX and $0.4 million for LifeMap Sciences.

Operating expenses for the full year 2019 were $14.0 million, as reported, which was comprised of $11.8 million for AgeX and $2.2 million for LifeMap Sciences, and were $11.2 million, as adjusted, comprised of $9.4 million for AgeX and $1.8 million for LifeMap Sciences.

Research and development expenses for the year ended December 31, 2019 decreased by $0.7 million to $5.9 million compared to $6.6 million in 2018. The decrease was mainly attributable to a nonrecurring expense of $800,000 to acquire certain in-process R&D in 2018.

General and administrative expenses for the year ended December 31, 2019 increased by $2.5 million to $8.1 million as compared with $5.6 million for 2018. The increases were mainly attributable to increased professional fees for consulting and accounting, insurance premiums, facilities related expenses, and non-cash stock-based compensation expense due to increased stock option grants. In April 2019 AgeX moved into its own facilities and terminated its shared facilities and services arrangement with Lineage Cell Therapeutics, Inc. (formerly BioTime, Inc.). Consequently AgeX now incurs the full cost of its facilities and finance and administrative personnel.

The reconciliation between operating expenses determined in accordance with accounting principles generally accepted in the United States (GAAP) and operating expenses, as adjusted, a non-GAAP measure, is provided in the financial tables included at the end of this press release.

Other income, net: Other income for the year ended December 31, 2019 was $0.3 million, as compared with $3.5 million in the same period in 2018. The decrease is entirely attributable to a nonrecurring $3.2 million gain on sale of our ownership interest in Ascendance Biotechnology, Inc. when that company was acquired by a third party in 2018.

Net loss attributable to AgeX: The net loss attributable to AgeX for the year ended December 31, 2019 was $12.2 million, or ($0.33) per share (basic and diluted) compared to $7.5 million, or ($0.21) per share (basic and diluted), for the same period in 2018.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan, and is seeking opportunities to establish licensing and collaboration arrangements around its broad IP estate and proprietary technology platforms.

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Certain statements contained in this release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the Risk Factors section of AgeXs most recent Annual Report on Form 10-K filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

AGEX THERAPEUTICS, INC. AND SUBSIDIARIES

CONSOLIDATED BALANCE SHEETS

(In thousands, except par value amounts)

December 31,

2019

2018

ASSETS

CURRENT ASSETS

Cash and cash equivalents

$

2,352

$

6,707

Accounts and grants receivable, net

363

131

Prepaid expenses and other current assets

1,339

1,015

Total current assets

4,054

7,853

Property and equipment, net

1,126

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AgeX Therapeutics Reports Fourth Quarter and Annual 2019 Financial Results and Provides Business Update - Business Wire

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1. Nettie Stevens (1861-1912, American) discovered that a fetuss sex is determined by chromosomes contributed by the parents during conception. Previously it was believed that the environmental factors during conception determined sex.

2. Charlotte Auerbach (1899-1994, German) has been called the mother of mutagenesis due to her discovery of genetic mutations caused by mustard gas. It only took her 2 months to discover these mutations in X chromosomes of males flies exposed to the gas. She received a Keith Prize in 1948.

3. Barbara McClintock (1902-1992, American) is known for her groundbreaking discovery of mobile genetic elements at the young age of 29, one of the greatest experiments of modern biology. For this work she was awarded the Nobel Prize in Physiology and Medicine in 1983.

4. Salome Gluecksohn-Waelsch (1907-2007, German) co-founded the field of developmental genetics. She used mouse embryo to study the effects of naturally occurring genetic mutations and the t-complex, a group of genes that direct the development of mouse tails. For all of her work in developmental genetics she was awarded the National Medal of Science in 1993 when she was 85 and still hard at work.

5. Rosalind Franklin (1920-1958, British) contribute to the major discovery of discovering the structure of DNA. She found that DNA takes two forms A and B. It was a photo of form B, photo 51, that provided Watson and Crick the the information that DNA was a double helix structure. Franklin also made major contributions on the filtration properties of types of coal during World War II. Franklin hypothesized that Tobacco Mosaic Virus was a hollow tube made of proteins that contained a single strand of RNA that spiraled inside the length of the tube like a thread spiraling inside a donut hole. After her early death at 38 from ovarian cancer, this hypothesis was found correct.

6. Esther Lederberg (1922-2006, American) provided foundation for future research in genetic inheritance in bacteria, gene regulation, and genetic recombination. She co-invented a simple method, replica plating, to reproduce bacterial colonies in masses while maintaining the original geometry of the colonies.

Photo credits top to bottom:The Marine Biological Laboratory,Jewish Studies,Encyclopdia Britannica,Albert Einstein College of Medicine,Nukas,Estherlederberg.com

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Two years after Ebola ravaged parts of West Africa, the deadly virus in 2018 was making a comeback in the Democratic Republic of the Congo.

Researchers at the University of Texas Medical Branch in Galveston sprang into action, reverse engineering the construction of a new vaccine and delivering 7,500 doses of it to the central African country for widespread use, all within 72 hours.

It was, in the words of Ben Raimer, interim president of UTMB, a proud moment for the university system, a collaborative effort that yielded life-saving results. Raimer cautioned, however, against the unrealistic expecations the Ebola success may have created for university researchers now grappling with the nuances of the far more complex novel coronavirus.

Were not a 72-hour virus maker here at UTMB, Raimer said. Weve done it one time for Ebola, but its not likely for this virus until we get a better understanding on how it functions in its various forms.

Coronavirus updates: Stay informed with accurate reporting you can trust

Public health experts generally predict that a coronavirus vaccine will take much longer and wont be ready for at least 12 to 18 months from the first known infection in late December. Whilemore than 20 vaccine candidates are in development, most are in the early stages, well before clinical trials. Uncertainty over the timeline has led to an unquenchable thirst for any morsel of good news regarding progress researchers have made in understanding how the virus attacks humans.

Scott Weaver understands this reality better than most. As the director of UTMBs infectious disease research programs, Weaver is tasked with helping manage nearly two-dozen projects related to the coronavirus, from macro initiatives like vaccine and antiviral treatment to more nuanced efforts such as why the virus affects people who smoke or vape more acutely.

For now, vaccine development is moving at a slower pace than Ebola, Weaver said, though he is hopeful that a previously developed SARS vaccine will prove effective. That vaccine, developed by researchers at Baylor College of Medicine and UTMB researchers, effectively protected mice against SARS, or severe acute respiratory syndrome, the pneumonia-causing virus from the same family a coronavirus that spread in the early 2000s. The vaccine never progressed to human testing because manufacturing of it wasnt completed until 2016, long after SARS had burned out.

Weaver noted two key challenges to completing work on the SARS vaccine: the genetically-altered laboratory mice used to test this vaccine had to be recreated from scratch; and funding sources, particularly from commercial interests, are hard to come by.

UTMB has cleared one hurdle. The transgenic mice embryos used for the original vaccine were recently implanted into female and male mice, and the first offspring were born several weeks ago. Of course, even after these mice are used to test vaccine candidates, those vaccines will have to be tested on non-human primates before the FDA will consider permission for clinical trials in people.

But even if UTMB does not win the vaccine arms race, the universitys coronavirus research has already made a significant difference in understanding the viruss complexities.

We have here three virologist faculty scientists who focus their work on coronavirus, so we were well prepared to gear up very quickly to do research on this virus, Weaver said.

Indeed, at the outset of the viral outbreak, UTMB developed a reverse genetic system to manipulate the virus genome. The Galveston National Laboratory at UTMB, a high-security biocontainment lab, was one of three labs in the country to get the coronavirus isolate in February after the Centers for Disease Control worked on the first virus sample in Washington state and cultured it in Atlanta.

Pei-Yong Shi, a professor of human genetics at UTMB, led this effort, which allows scientists to essentially recreate the virus from scratch.

We can understand the mutations and history of the virus. We will be able to manipulate the virus, to understand which regions are causing the disease so we can make vaccines and therapeutics, Shi said.

The UTMB genetic system played a vital role in helping develop badly needed diagnostic tests. The universitys World Reference Center for Emerging Viruses and Arboviruses stockpiled the viral RNA the genetic material needed to optimize tests for federal approval.

There was a time period in late February where we were literally the only laboratory in the world providing these RNA samples for diagnostic (test) development, Weaver said. A lot of the big companies that you see now are starting to scale up diagnostics - like LabCorps and Quest and many of the big hospitals including some in Houston and here in Galveston - we provided that critical RNA to them so they could get their tests up and running as quick as possible.

One of the primary projects capturing the attention of UTMB scientists is testing antiviral drugs to treat the symptoms of the coronavirus, Weaver said. The drugs currently being tested were developed for other viral infections or non-infectious diseases, such as remdesivir, which was used to combat Ebola infections.

Both President Trump and the World Health Organization have highlighted remdesivir as a promising coronavirus treatment, though clinical trials are still ongoing to determine how effective the drug can be. UTMB has a clinical trial set up in the coming weeks to test remdesivir in Galveston County coronavirus patients.

Coronavirus in Houston: All of the latest news, numbers and analysis to keep you up-to-date, only on HoustonChronicle.com

In the middle of an outbreak like this, there are going to be so many people who are hospitalized and eligible for these clinical trials that well learn very quickly whether (remdesivir) has efficacy or not, Weaver said. I think thats really the best prospect for an improvement in patient care in the near future.

Tapping into funding sources to continue vaccine research is a bigger problem. One of the major differences between Ebola in 2018 and coronavirus that contributed to how quickly UTMB was able to develop a vaccine was the sustained funding for Ebola research. Besides the SARS outbreak in the early aughts and MERS in 2012, coronaviruses typically dont attract the same interest.

Its much harder to get funding, especially commercial interest, in the coronavirus vaccine, Weaver said. Unfortunately, that means we dont have as much to start from. There were some vaccines that were developed. They never went very far down the pipeline towards clinical trials, but at least were not starting completely from scratch.

But for as much work is being done behind the scenes in the race to cure and treat the coronavirus, Weaver said the immediate outcome and toll of this pandemic will be determined by public health measures such as social distancing.

One person on average transmits the virus to 3 or 4 additional people and if one of those is a high-risk person, they may die, if one of those is a healthcare worker, they may spread it to many more people, Weaver said. I just hope that everyone takes this very seriously.

nick.powell@chron.com

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Researchers at Stanford University report that they can rejuvenate human cells by reprogramming them back to a youthful state. They hope that the technique will help in the treatment of diseases, such as osteoarthritis and muscle wasting, that are caused by the aging of tissue cells.

A major cause of aging is thought to be the errors that accumulate in the epigenome, the system of proteins that packages the DNA and controls access to its genes. The Stanford team, led by Tapash Jay Sarkar, Dr. Thomas A. Rando and Vittorio Sebastiano, say their method, designed to reverse these errors and walk back the cells to their youthful state, does indeed restore the cells vigor and eliminate signs of aging.

In their report, published on Tuesday in Nature Communications, they described their technique as a significant step toward the goal of reversing cellular aging and could produce therapies for aging and aging-related diseases.

Leonard P. Guarente, an expert on aging at M.I.T., said the method was one of the most promising areas of aging research but that it would take a long time to develop drugs based on RNA, the required chemical.

The Stanford approach utilizes powerful agents known as Yamanaka factors, which reprogram a cells epigenome to its time zero, or embryonic state.

Embryonic cells, derived from the fertilized egg, can develop into any of the specialized cell types of the body. Their fate, whether to become a skin or eye or liver cell, is determined by chemical groups, or marks, that are tagged on to their epigenome.

In each type of cell, these marks make accessible only the genes that the cell type needs, while locking down all other genes in the DNAs. The pattern of marks thus establishes each cells identity.

As the cell ages, it accumulates errors in the marking system, which degrade the cells efficiency at switching on and off the genes needed for its operations.

In 2006 Dr. Shinya Yamanaka, a stem-cell researcher at Kyoto University, amazed biologists by showing that a cells fate could be reversed with a set of four transcription factors agents that activate genes that he had identified. A cell dosed with the Yamanaka factors erases the marks on the epigenome, so the cell loses its identity and reverts to the embryonic state. Erroneous marks gathered during aging are also lost in the process, restoring the cell to its state of youth. Dr. Yamanaka shared the 2012 Nobel Prize in medicine for the work.

But the Yamanaka factors are no simple panacea. Applied to whole mice, the factors made cells lose their functions and primed them for rapid growth, usually cancerous; the mice all died.

In 2016, Juan Carlos Izpisua Belmonte, of the Salk Institute for Biological Studies in San Diego, found that the two effects of the Yamanaka factors erasing cell identity and reversing aging could be separated, with a lower dose securing just age reversal. But he achieved this by genetically engineering mice, a technique not usable in people.

In their paper on Tuesday, the Stanford team described a feasible way to deliver Yamanaka factors to cells taken from patients, by dosing cells kept in cultures with small amounts of the factors.

If dosed for a short enough time, the team reported, the cells retained their identity but returned to a youthful state, as judged by several measures of cell vigor.

Dr. Sebastiano said the Yamanaka factors appeared to operate in two stages, as if they were raising the epigenomes energy to one level, at which the marks of aging were lost, and then to a higher level at which cell identity was erased.

The Stanford team extracted aged cartilage cells from patients with osteoarthritis and found that after a low dosage of Yamanaka factors the cells no longer secreted the inflammatory factors that provoke the disease. The team also found that human muscle stem cells, which are impaired in a muscle-wasting disease, could be restored to youth. Members of the Stanford team have formed a company, Turn Biotechnologies, to develop therapies for osteoarthritis and other diseases.

The study is definitively a step forward in the goal of reversing cellular aging, Dr. Izpisua Belmonte said.

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Dr. Jim Surrell, Journal columnist

The overall function of our human immune system is to prevent or limit infection. The primary job of our immune system is to distinguish between our normal, healthy cells and possible other dangerous cells, such as viruses and bacteria that may come into our blood stream. Our immune system is always on duty to look for and recognize these possible infectious viruses and bacteria.

The immune system looks closely at these potentially infectious cells to do all it can to prevent us from getting an infection.

Know that we rely on our immune system every day to help us fight off infections and keep us healthy. Our immune system contains numerous cell types that either circulate throughout the body or reside in our particular body tissues. Each cell type plays a unique role, with different ways of performing their function to fight off infections. Further, our great medical and scientific researchers continuously work to optimize our immune responses to confront specific potential infectious issues, such as is being done at this time with regard to the coronavirus (COVID-19).

Let us now take a look at the many components of our human immune system and how our body works to fight off infections. All of our numerous immune cells come from basic immune cells in our bone marrow and develop into mature cells through a series of changes that can occur in different parts of the body. Following is a brief look at our various body components that make up our immune system.

Skin: Our skin is usually the first line of defense against infectious organisms. Skin cells produce and secrete important antimicrobial proteins, and immune cells can be found in specific layers of our skin.

Bone marrow: Our bone marrow contains stem cells that can develop into a variety of cell types. These stem cells in our bone marrow develop our many various types of immune cells that are very important first-line responders to infection. These stem cells create our essential infection-fighting cells, called B cells and T cells. These B cells and T cells are responsible for mounting a response to specific microbes that may cause infections. We also have natural niller (NK) immune cells that also provide defenses to fight off infections. These immune system B cells, T cells, and NK cells are also called lymphocytes.

Bloodstream: Immune cells constantly circulate throughout the bloodstream, patrolling for problems. When blood tests are used to monitor white blood cells, another term for immune cells, a snapshot of the immune system is taken. If our white blood cells are too few, or overabundant in the bloodstream, this may reflect a problem that should be addressed by a professional health care provider.

Thymus Gland: Immune system T cells mature in our small thymus gland, located in the upper chest.

Lymphatic system: The lymphatic system is a network of vessels and tissues composed of lymph, an extracellular fluid, and our lymph nodes. The lymphatic system is the essential part of our immune system that provides communication between our various body tissues and our bloodstream. Immune cells are carried through the lymphatic system and converge in lymph nodes. Lymph nodes are a communication hub where immune cells sample information brought in from the body. Thus, doctors may check patients for swollen lymph nodes, which may indicate an active immune response.

Spleen: The spleen is an organ located behind the stomach. Immune cells are found in the spleen and if there are any blood-borne infectious organisms, these immune cells activate and respond accordingly.

Lastly, be aware that our human immune system regenerates and repairs itself every night when we sleep. Studies show that people who dont get quality sleep or enough sleep are more likely to get sick after being exposed to an infectious virus or bacteria. Lack of sleep can also affect how fast you recover if you do get sick. To keep you and your immune system healthy, get the recommended seven to eight hours of sleep each and every night. And yes, these are Doctors Orders!

EDITORS NOTE: Dr. Jim Surrell is the author of The ABCs For Success In All We Do and the SOS (Stop Only Sugar) Diet books. Requests for health topics for this column are encouraged. Contact Dr. Surrell by email at sosdietdoc@gmail.com.

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TORONTO, March 31, 2020 /CNW/ - The Gairdner Foundation is pleased to announce the 2020 Canada Gairdner Award laureates, recognizing some of the world's most significant biomedical research and discoveries. During these challenging times, we believe it is important to celebrate scientists and innovators from around the world and commend them for their tireless efforts to conduct research that impacts human health.

2020 Canada Gairdner International AwardThe five 2020 Canada Gairdner International Award laureates are recognized for seminal discoveries or contributions to biomedical science:

Dr. Masatoshi TakeichiSenior Visiting Scientist, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan; Professor Emeritus, Kyoto University, Kyoto, Japan

Dr. Rolf KemlerEmeritus Member and Director, Max Planck Institute of Immunobiology and Epigenetics, Freiburg, Germany

Awarded "For their discovery, characterization and biology of cadherins and associated proteins in animal cell adhesion and signalling."

Dr. Takeichi

The Work: The animal body is made up of numerous cells. Dr. Takeichi was investigatinghow animal cells stick together to form tissues and organs, and identified a key protein which he named 'cadherin'.Cadherin is present on the surface of a cell and binds to the same cadherin protein on the surface of another cell through like-like interaction, thereby binding the cells together. Without cadherin, cell to cell adhesion becomes weakened and leads to the disorganization of tissues. Dr. Takeichi found that there are multiple kinds of cadherin within the body, each of which are made by different cell types, such as epithelial and neuronal cells. Cells with the same cadherins tend to cluster together, explaining the mechanism of how different cells are sorted out and organized to form functional organs.

Further studies by Dr. Takeichi's group showed that cadherin function is supported by a number of cytoplasmic proteins, includingcatenins, and their cooperation is essential for shaping of tissues. His studies also revealed that the cadherin-dependent adhesion mechanism is involved in synaptic connections between neurons, which are important for brain wiring.

Dr. Kemler

The Work: Dr. Kemler, using an immunological approach, developed antibodies directed against surface antigens of early mouse embryos. These antibodies were shown to prevent compaction of the mouse embryo and interfered with subsequent development. Both Dr. Kemler and Dr. Takeichi went on to clone and sequence the gene encoding E-cadherin and demonstrate that it was governing homophilic cell adhesion.

Dr. Kemler also discovered the other proteins that interact with the cadherins, especially the catenins, to generate the machinery involved in animal cell-to-cell adhesion. This provided the first evidence of their importance in normal development and diseases such as cancer. It has been discovered that cadherins and catenins are correlated to the formation and growth of some cancers and how tumors continue to grow. Beta catenin is linked to cell adhesion through interaction with cadherins but is also a key component of the Wnt signalling pathway that is involved in normal development and cancer. There are approximately 100 types of cadherins, known as the cadherin superfamily.

Dr. Takeichi

The Impact: The discovery of cadherins, which are found in all multicellular animalspecies, has allowed us to interpret how multicellular systems are generated and regulated. Loss of cadherin function has been implicated as the cause of certain cancers, as well as in invasiveness of many cancers. Mutations in special types of cadherin result in neurological disorders, such as epilepsy and hearing loss. The knowledge of cadherin function is expected to contribute to the development of effective treatments against such diseases.

Dr. Kemler

The Impact: Human tumors are often of epithelial origin. Given the role of E-cadherin for the integrity of an epithelial cell layer, the protein can be considered as a suppressor of tumor growth. The research on the cadherin superfamily has had great impact on fields as diverse as developmental biology, cell biology, oncology, immunology and neuroscience. Mutations in cadherins/catenins are frequently found in tumors. Various screens are being used to identify small molecules that might restore cell adhesion as a potential cancer therapy.

Dr. Roel NusseProfessor & Chair, Department of Developmental Biology; Member, Institute for StemCell Biology andRegenerativeMedicine, Stanford University, School of Medicine.Virginia and Daniel K. Ludwig Professor of Cancer Research. Investigator, Howard Hughes Medical Institute

Awarded"For pioneering work on the Wnt signaling pathway and its importance in development, cancer and stem cells"

The Work: Dr. Nusse's research has elucidated the mechanism and role of Wnt signaling, one of the most important signaling systems in development. There is now abundant evidence that Wnt signaling is active in cancer and in control of proliferation versus differentiation of adult stem cells, making the Wnt pathway one of the paradigms for the fundamental connections between normal development and cancer.

Among Dr. Nusse's contributions is the original discovery of the first Wnt gene (together with Harold Varmus) as an oncogene in mouse breast cancer. Afterwards Dr. Nusse identified the Drosophila Wnt homolog as a key developmental gene, Wingless. This led to the general realization of the remarkable links between normal development and cancer, now one of the main themes in cancer research. Using Drosophila genetics, he established the function of beta-catenin as a mediator of Wnt signaling and the Frizzleds as Wnt receptors (with Jeremy Nathans), thereby establishing core elements of what is now called the Wnt pathway. A major later accomplishment of his group was the first successful purification of active Wnt proteins, showing that they are lipid-modified and act as stem cell growth factors.

The Impact: Wnt signaling is implicated in the growth of human embryos and the maintenance of tissues. Consequently, elucidating the Wnt pathway is leading to deeper insights into degenerative diseases and the development of new therapeutics. The widespread role of Wnt signaling in cancer is significant for the treatment of the disease as well. Isolating active Wnt proteins has led to the use of Wnts by researchers world-wide as stem cell growth factors and the expansion of stem cells into organ-like structures (organoids).

Dr. Mina J. Bissell Distinguished Senior Scientist, Biological Systems and Engineering Division, Lawrence Berkeley National Laboratory; Faculty; Graduate Groups in Comparative Biochemistry, Endocrinology, Molecular Toxicology and Bioengineering, University of California Berkeley, Berkeley, CA, USA

Awarded "For characterizing "Dynamic Reciprocity" and the significant role that extracellular matrix (ECM) signaling and microenvironment play in gene regulation in normal and malignant cells, revolutionizing the fields of oncology and tissue homeostasis."

The Work: Dr. Mina Bissell's career has been driven by challenging established paradigms in cellular and developmental biology. Through her research, Dr. Bissell showed that tissue architecture plays a dominant role in determining cell and tissue phenotype and proposed the model of 'dynamic reciprocity' (DR) between the extracellular matrix (ECM) and chromatin within the cell nucleus. Dynamic reciprocity refers to the ongoing, bidirectional interaction between cells and their microenvironment. She demonstrated that the ECM could regulate gene expression just as gene expression could regulate ECM, and that these two phenomena could occur concurrently in normal or diseased tissue.

She also developed 3D culture systems to study the interaction of the microenvironment and tissue organization and growth, using the mammary gland as a model.

The Impact:Dr. Bissell's model of dynamic reciprocity has been proven and thoroughly established since its proposal three decades ago and the implications have permeated every area of cell and cancer biology, with significant implications for current and future therapies. Dr. Bissell's work has generated a fundamental and translationally crucial paradigm shift in our understanding of both normal and malignant tissues.

Her findings have had profound implications for cancer therapy by demonstrating that tumor cells can be influenced by their environment and are not just the product of their genetic mutations. For example, cells from the mammary glands grown in two-dimensional tissue cultures rapidly lose their identity, but once placed in proper three-dimensional microenvironments, they regain mammary form and function. This work presages the current excitement about generation of 3D tissue organoids and demonstrates Dr. Bissell's creative and innovative approach to science.

Dr. Elaine FuchsHoward Hughes Medical Institute Investigator and Rebecca C. Lancefield Professor and Head of the Robin Chemers Neustein Laboratory of Mammalian Cell Biology and Cell Biology; The Rockefeller University, New York, NY, USA

Awarded"For her studies elucidating the role of tissue stem cells in homeostasis, wound repair, inflammation and cancer."

The Work: Dr. Fuchs has used skin to study how the tissues of our body are able to replace dying cells and repair wounds. The skin must replenish itself constantly to protect against dehydration and harmful microbes. In her research, Fuchs showed that this is accomplished by a resident population of adult stem cells that continually generates a shell of indestructible cells that cover our body surface.

In her early research, Fuchs identified the proteins---keratinsthat produce the iron framework of the skin's building blocks, and showed that mutations in keratins are responsible for a group of blistering diseases in humans. In her later work, Fuchs identified the signals that prompt skin stem cells to make tissue and when to stop. In studying these processes, Fuchs learned that cancers hijack the fundamental mechanisms that tissue stem cells use to repair wounds. Her team pursued this parallel and isolated and characterized the malignant stem cells that are responsible for propagating a type of cancer called "squamous cell carcinoma." In her most recent work, she showed that these cells can be resistant to chemotherapies and immunotherapies and lead to tumor relapse.

The Impact: All tissues of our body must be able to replace dying cells and repair local wounds. Skin is particularly adept at performing these tasks. The identification and characterization of the resident skin stem cells that make and replenish the epidermis, sweat glands and hair provide important insights into this fountain of youth process and hold promise for regenerative medicine and aging. In normal tissues, the self-renewing ability of stem cells to proliferate is held in check by local inhibitory signals coming from the stem cells' neighbours. In injury, stimulatory signals mobilize the stem cells to proliferate and repair the wound. In aging, these normal balancing cues are tipped in favour of quiescence. In inflammatory disorders, stem cells become hyperactivated. In cancers, the wound mechanisms to mobilize stem cells are hijacked, leading to uncontrolled tissue growth. Understanding the basic mechanisms controlling stem cells in their native tissue is providing new strategies for searching out refractory tumor cells in cancer and for restoring normalcy in inflammatory conditions.

2020 John Dirks Canada Gairdner Global Health AwardThe 2020 John Dirks Canada Gairdner Global Health Award laureate is recognized for outstanding achievements in global health research:

Professor Salim S. Abdool KarimDirector of CAPRISA (Centre for the AIDS Program of Research in South Africa), the CAPRISA Professor in Global Health at Columbia University, New York and Pro Vice-Chancellor (Research) at the University of KwaZulu-Natal, Durban, South Africa

Professor Quarraisha Abdool KarimAssociate Scientific Director of CAPRISA, Professor in Clinical Epidemiology, Columbia University, New York and Professor in Public Health at the Nelson Mandela Medical School and Pro Vice-Chancellor (African Health) at the University of KwaZulu-Natal, Durban, South Africa

Awarded"For their discovery that antiretrovirals prevent sexual transmission of HIV, which laid the foundations for pre-exposure prophylaxis (PrEP), the HIV prevention strategy that is contributing to the reduction of HIV infection in Africa and around the world."

The Work: UNAIDS estimates that 37 million people were living with HIV and 1.8 million people acquired HIV in 2017. In Africa, which has over two thirds of all people with HIV, adolescent girls and young women have the highest rates of new HIV infections. ABC (Abstinence, Be faithful, and use Condoms) prevention messages have had little impact - due to gender power imbalances, young women are often unable to successfully negotiate condom use, insist on mutual monogamy, or convince their male partners to have an HIV test.

In responding to this crisis, Salim and Quarraisha Abdool Karim started investigating new HIV prevention technologies for women about 30 years ago. After two unsuccessful decades, their perseverance paid off when they provided proof-of-concept that antiretrovirals prevent sexually acquired HIV infection in women. Their ground-breaking CAPRISA 004 trial showed that tenofovir gel prevents both HIV infection and genital herpes. The finding was ranked inthe "Top 10 Scientific Breakthroughs of 2010" by the journal, Science. The finding was heralded by UNAIDS and the World Health Organization (WHO) as one of the most significant scientific breakthroughs in AIDS and provided the first evidence for what is today known as HIV pre-exposure prophylaxis (PrEP).

The Abdool Karims have also elucidated the evolving nature of the HIV epidemic in Africa, characterising the key social, behavioural and biological risk factors responsible for the disproportionately high HIV burden in young women. Their identification of the "Cycle of HIV Transmission", where teenage girls acquire HIV from men about 10 years older on average, has shaped UNAIDS policies on HIV prevention in Africa.

The impact: CAPRISA 004 and several clinical trials of oral tenofovir led tothe WHO recommending a daily tenofovir-containing pill for PrEP as a standard HIV prevention tool for all those at high risk a few years later. Several African countries are among the 68 countries across all continents that are currently making PrEP available for HIV prevention. The research undertaken in Africa by this South African couple has played a key role in shaping the local and global response to the HIV epidemic.

2020 Canada Gairdner Wightman AwardThe 2020 Canada Gairdner Wightman Award laureate is a Canadian scientist recognized for outstanding leadership in medicine and medical science throughout their career:

Dr. Guy Rouleau Director of the Montreal Neurological Institute-Hospital (The Neuro); Professor & Chair of the Department of Neurology and Neurosurgery, McGill University; Director of the Department of Neuroscience, McGill University Health Center

Awarded "For identifying and elucidating the genetic architecture of neurological and psychiatric diseases, including ALS, autism and schizophrenia, and his leadership in the field of Open Science."

The Work: Dr. Rouleau has identified over 20 genetic risk factors predisposing to a range of brain disorders, both neurological and psychiatric, involving either neurodevelopmental processes or degenerative events. He has defined a novel disease mechanism for diseases related to repeat expansions that are at play in some of the most severe neurodegenerative conditions. He has significantly contributed to the understanding of the role of de novo variants in autism and schizophrenia. In addition, he has made important advances for various neuropathies, in particular for amyotrophic lateral sclerosis (ALS) where he was involved in the identification of the most prevalent genetic risk factors -which in turn are now the core of innumerable ALS studies worldwide.

Dr. Rouleau has also played a pioneering role in the practice of Open Science (OS), transforming the Montreal Neurological Institute-Hospital (The Neuro) into the first OS institution in the world. The Neuro now uses OS principles to transform research and careand accelerate the development of new treatments for patients through Open Access, Open Data, Open Biobanking, Open Early Drug Discovery and non-restrictive intellectual property.

The Impact: The identification of genetic risk factors has a number of significant consequences. First, allowing for more accurate genetic counselling, which reduces the burden of disease to affected individuals, parents and society. A revealing case is Andermann syndrome, a severe neurodevelopmental and neurodegenerative condition that was once relatively common in the Saguenay-Lac-St-Jean region of Quebec. Now this disease has almost disappeared from that population. Second, identifying the causative gene allows the development of treatments. For instance, his earlier work on a form of ALS linked to the superoxide dismutase-1 gene (SOD1) opened up studies which are now the focal point of phase 2 clinical studies showing great promise.

Byactingasalivinglabforthelast coupleofyears,TheNeuroisspearheading the practice of OpenScience (OS).TheNeurois alsoengagingstakeholdersacross Canadawiththegoal of formalizinganational OSallianceforthe neurosciences.Dr.Rouleau'sworkinOScontributesfundamentallytothetransformationoftheveryecosystemofsciencebystimulatingnewthinkingandfosteringcommunitiesofsharing.InspiredbyTheNeuro'svision,theglobalsciencecommunityisreflecting oncurrentresearchconventionsandcollaborativeprojects,andthemomentumforOSisgainingafootholdinorganizationsandinstitutionsinallcornersoftheearth.

About the Gairdner Foundation:

The Gairdner Foundation was established in 1957 by Toronto stockbroker, James Gairdner to award annual prizes to scientists whose discoveries have had major impact on scientific progress and on human health. Since 1959 when the first awards were granted, 387scientists have received a Canada Gairdner Award and 92 to date have gone on to receive the Nobel Prize.The Canada Gairdner Awards promote a stronger culture of research and innovation across the country through our Outreach Programs including lectures and research symposia. The programs bring current and past laureates to a minimum of 15 universities across Canada to speak with faculty, trainees and high school students to inspire the next generation of researchers. Annual research symposia and public lectures are organized across Canada to provide Canadians access to leading science through Gairdner's convening power.

http://www.gairdner.org

SOURCE Gairdner Foundation

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2020 Canada Gairdner Awards Recognize World-renowned Scientists for Transformative Contributions to Research That Impact Human Health - Benzinga

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They say its the little things that matter, and when it comes to skincare, we have to agree. Few things feel better than the sensation of a freshly scrubbed face. Trouble is, few of us know how to actually get that feeling consistently. Sure, you may know that the key to a clear, healthy complexion is exfoliation (i.e., shedding the uppermost layer of dead skin cells). But what you use to exfoliate makes a big difference in whether you come away with a radiant glow or a mask of raw, irritated epidermis.

There are a few types of products that will do the job, but exfoliating face scrubs are often the most preferred. Known as physical exfoliants (as opposed to chemical exfoliants), these facial scrubs use tiny abrasive grains that act like sandpaper on your face, using friction to buff dead skin cells away. Physical exfoliants can be made from natural ingredients like apricot kernels, sugar, even coffee, or they can be synthetic beads that dissolve as you use them. Some guys love this type of exfoliant because you can really feel it working. However, it can be pretty harsh on some sensitive skin, leaving your face red and irritated, which leads to dry skin, increased oil production, or even scarring (and not in a sexy Jason Momoa kind of way).

That doesnt mean you shouldnt use an exfoliating scrub. It just means you have to do your homework or, if youd rather, have us do it for you. Weve put together a list of our favorite exfoliating face scrubs that contain quality ingredients for buffing away dead skin, and soothing the new skin underneath so that it stays happy, hydrated, and balanced. Gently (and we mean gently) massage these facial exfoliator scrubs into your forehead, chin, and cheeks, splash with cold water, pat dry, and finish with a kiss of moisturizer, and youll feel your face glowing all day long.

Finely ground apricot seed sloughs away yesterdays skin, with a little extra help from papaya extract enzymes, while chamomile, aloe, and sea kelp nourish and hydrate whats underneath.

For nights when youre ready to do some deep cleaning, pick this ultra-pure exfoliating mask. Activated charcoal and bentonite clay absorb impurities from your skin while the mask remains on your face. Scrubbing it off leaves your face not only clear, but also unbelievably soft, thanks to the addition of willow bark, rosemary extract, and sulfur mud.

Ground walnut shells and bamboo stem polish your skin, while jojoba esters and tropical fruit extracts break down pore-clogging oils, and special hydrating sugar technology blend rebuilds your skins moisture barrier before you rinse clean.

This super-concentrated, double-action exfoliating face scrub doesnt play around. Tiny jojoba beads offer a physical exfoliation that is boosted with the chemical exfoliating power of glycolic and salicylic acid. Five minutes with this scrub, and youll look and feel like a new man.

Sometimes, the simplest solutions are the best. This easy-to-find face scrub boasts natural ingredients such as aloe, hemp, and other nutrient-rich botanicals that ensure your newly scrubbed skin doesnt get dry or irritated.

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Give Your Face a Spring Refresh With These Exfoliating Face Scrubs - The Manual

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DetailsCategory: DNA RNA and CellsPublished on Tuesday, 31 March 2020 10:51Hits: 317

CAMBRIDGE, MA & SAN FRANCISCO, CA, USA I March 30, 2020 IGeneration Bio and Vir Biotechnology (Nasdaq: VIR) today announced a collaborative research agreement to explore the potential for Generation Bios non-viral gene therapy platform to extend the impact and reach of Virs current or future human monoclonal antibodies (mAb) against SARS-CoV-2, the virus responsible for COVID-19. Generation Bios technology has the potential to deliver genetic information directly to cells without the use of adeno-associated viruses (AAV), in effect instructing the patients body to produce the antibody itself.

The companies believe that this technology, coupled with Virs potent neutralizing antibodies, has the potential to provide effective, long-lasting protection against SARS-CoV-2. Virs leading antibody was isolated from a SARS-CoV-1 recovered patient and potently neutralizes SARS-CoV-2. Vir believes that this approach can potentially provide broad and longer-lasting protection.

Together, we believe we can develop long-lasting therapies suitable for population-wide prevention and treatment, said Generation Bio President and CEO Geoff McDonough, M.D. We are moving with urgency to explore leveraging our platform to build protection against COVID-19 for the long term.

Generation Bios proprietary non-viral gene therapy platform is designed to enable production of target proteins from a patients own cells. This approach may allow the patient to maintain stable levels of antibody expression for years, providing continuous protection against the target virus. In addition, the companies intend to leverage Generation Bios scalable manufacturing process to potentially extend the reach of Virs monoclonal antibodies to a greater number of patients.

We are eager to bring our antibodies to patients as quickly as possible, and should they work, to make them available to as many patients as quickly as possible. We are excited to explore the potential of ceDNA in an infectious disease setting and our anti-SARS-CoV-2 program offers a way to do that, said George Scangos, Ph.D., CEO of Vir. Both companies are highly motivated to make meaningful contributions to stopping this disease and we look forward to a productive collaboration with Generation Bio.

About Generation Bio

Generation Bio is an innovative genetic medicines company focused on creating a new class of gene therapy to provide durable, redosable treatments for patients suffering from both rare and prevalent diseases. The companys non-viral platform incorporates a proprietary high-capacity DNA construct called closed-ended DNA, or ceDNA; a novel cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The company is designing therapies to provide targeted delivery of genetic payloads that include large and multiple genes across a broad array of tissues, and to be redosable for individualized and extended treatment throughout a patients life. The platform is designed to expand access to treatments for rare diseases and to address prevalent diseases through efficient, scalable manufacturing.

About Virs Antibody Platform

Vir has a robust method for capitalizing on unusually successful immune responses naturally occurring in people who are protected from, or have recovered from, infectious diseases. The platform is used to identify rare antibodies from survivors that have the potential to treat and prevent rapidly evolving and/or previously untreatable pathogens via direct pathogen neutralization and immune system stimulation. Vir engineers the fully human antibodies that it discovers to enhance their therapeutic potential. This platform has been used to identify and develop antibodies for pathogens including Ebola (mAb114, currently in use in the Democratic Republic of Congo), hepatitis B virus, influenza A, SARS-CoV-2, malaria, and others.

About Vir Biotechnology

Vir Biotechnology is a clinical-stage immunology company focused on combining immunologic insights with cutting-edge technologies to treat and prevent serious infectious diseases. Vir has assembled four technology platforms that are designed to stimulate and enhance the immune system by exploiting critical observations of natural immune processes. Its current development pipeline consists of product candidates targeting hepatitis B virus, influenza A, SARS-CoV-2, human immunodeficiency virus, and tuberculosis. For more information, please visit http://www.vir.bio.

SOURCE: Generation Bio

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Generation Bio and Vir Biotechnology to Collaborate on Research to Leverage Scalable Non-Viral Gene Therapy Platform for Durable Production of...

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A programme investigating the potential of gene therapy for the treatment of osteoarthritis in horses, dogs and cats, has begun at the Royal Veterinary College (RVC), led by Skeletal Regeneration expert, Dr Scott Roberts.

Dr Scott Roberts, who joined the RVC as Senior Lecturer in Translational Skeletal Research in the Department of Comparative Biomedical Sciences, has recently initiated the programme of investigative research.

The research is inspired by Dr Roberts interest in skeletal stem cell signalling and the effect of signal modulation on skeletal tissue regeneration. He initiated this work during his time at KU Leuven (Belgium) but has subsequently researched this topic as Principal Investigator at University College London and as Senior Principal Scientist at UCB Pharma. During his time at UCB, Dr Roberts identified musculoskeletal drug targets and led the subsequent drug discovery projects. His research has resulted in several patent applications on the use of cells and therapeutic antibodies to promote the regeneration of bone and cartilage.

The new gene therapy research programme in osteoarthritis will be undertaken in partnership with the LEP funded Vaccinology and Cell Therapy Hub at the RVC. This will not only provide an environment for further research on regenerative medicine but also the facilities to create reagents for clinical translation.

The Hub with its close connections to both scientists and veterinary clinicians is also well-placed to take the science from bench-to-bedside and facilitate clinical trials in veterinary patients. This will include horses at the Equine Referral Hospital, and dogs and cats at the Queen Mother Hospital for Animals at the RVC.

It is proposed that this treatment would transition into human clinical studies, exemplifying the RVCs commitment to the One Health approach which recognises and facilitates the synergy between animal and human health.

Dr Scott Roberts said:

This research has the potential to change the way that we approach degenerative joint disease and I am delighted to have access to the Vaccinology and Cell Therapy Hub while we undertake this work. We hope that this science will lead to a ground-breaking treatment for osteoarthritis in animals, and eventually humans. We are optimistic about the future of this research, particularly given its contribution to the RVCs One Health ethos. I look forward to taking advantage of the RVCs world-leading facilities, as well as the scientists and clinicians who will help us take this research forward.

Professor Jonathan Elliott, Vice Principal of Research and Innovation at the RVC, said:

We are delighted that Scott Roberts has joined the RVC and will pursue his translational research into novel therapies that stimulate repair of cartilage for osteoarthritis in horses, dogs and people. This appointment builds on the RVCs expertise in Skeletal Biology. Scotts links to the Pharma Industry are very important for his translational science to have impact. His work fits perfectly with the goals of our soon to open Vaccinology and Cell Therapy Hub, made possible by the funding from the Hertfordshire Local Enterprise Partnership.

The research Dr Roberts is undertaking is critical as cartilage has a very poor capacity to repair itself, with cartilage injuries often progressing to osteoarthritis. There is also currently no approved evidence-driven therapy for the treatment of this disease.

In addition to osteoarthritis, Dr Roberts research aims to create regenerative therapeutics for non-healing bone fractures. This is based on a comprehensive understanding of tissue development, as tissue repair is now regarded as a re-emergence of embryonic signalling cascades. Dr Roberts has used knowledge in this area to identify developmentally inspired methodologies to create laboratory grown tissue implants that have the capacity to drive bone fracture repair.

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Investigation Begins into Gene Therapy for Osteoarthritis - Everything Horse UK

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Covington-based CTIClinical Trial and Consulting Services has partnered with California-based Humanigen, Inc. on a study for the treatment of novel coronavirus 2019 (COVID-19).

Humanigen, Inc., (HGEN) (Humanigen), a clinical-stage biopharmaceutical company focused on preventing and treating cytokine storm with lenzilumab, the companys proprietary Humaneered anti-human-granulocyte-macrophage colony-stimulating factor (GM-CSF) monoclonal antibody, announced that the company has submitted an initial protocol synopsis to the FDA in support of the companys plans to initiate a multi-center, US, Phase III study in COVID-19 patients.

The study, if approved, will be a randomized, controlled, clinical trial with lenzilumab for the prevention of ARDS and/or death in hospitalized patients with pneumonia associated with coronavirus 2 (SARS-CoV-2) infection in COVID-19.

The study will be conducted in partnership with CTI, which specializes in complex programs for critically and chronically ill patients.

Humanigen, Inc. is developing its portfolio of clinical and pre-clinical therapies for the treatment of cancers and infectious diseases via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms.

Humanigen has pioneered the field of GM-CSF neutralization and, unlike others, has already conducted two Phase I and two Phase II studies, including in patients with severe respiratory conditions, with excellent safety results, said Dr. Cameron Durrant, chief executive officer of Humanigen. We have been working on prevention of cytokine storm for nearly three years. Lenzilumab has an excellent safety and tolerability profile and has not been associated with serious adverse events, including in patients who are immunosuppressed or with severe asthma. Subject to discussion and agreement with regulatory authorities, we intend to progress as quickly as possible with this study.

More details on the companys programs in COVID-19 can be found on the companys websiteunder the COVID-19 tab.

CTI Clinical Trial and Consulting Services is a global, privately held, full-service contract research organization (CRO).

It delivers a complete spectrum of clinical trial and consulting services throughout the lifecycle of development, from concept to commercialization.

CTIs focused therapeutic approach provides pharmaceutical, biotechnology, and medical device firms with clinical and disease area expertise in rare diseases, regenerative medicine/gene therapy, immunology, transplantation, nephrology, hematology/oncology, neurology, infectious diseases, hepatology, cardiopulmonary, and pediatric populations.

Schroeder

The COVID-19 pandemic demands an unprecedented response from the pharmaceutical and biotechnology communities, requiring innovation, collaboration, and prioritization of speed without the cost of safety, remarked Tim Schroeder, founder and CEO of CTI. We are proud to have the opportunity to collaborate on such a response with the Humanigen team and to work together to develop an effective treatment solution for those hardest hit by COVID-19.

CTI also offers a fully integrated multi-specialty clinical research site that conducts phase I-IV trials. CTI has a passion for helping life-changing therapies succeed in chronically and critically ill patient populations. With clinical trial experience across 6 continents, CTI partners with research sites, patients, and sponsors to fulfill unmet medical needs. CTI is headquartered in the Greater Cincinnati, OH area, with operations across North America, Europe, Latin America, and Asia-Pacific.

For more information on CTI, click here.

Study to tackle leading cause of death in COVID-19 patients

Lenzilumab, a monoclonal antibody that neutralizes granulocyte macrophage colony stimulating factor (GM-CSF)

GM-CSF is up-regulated in COVID-19 patients and may cause a harmful immune response leading to acute respiratory distress syndrome (ARDS)

Clinical evidence suggests that this harmful immune response contributes to cytokine storm in COVID-19 patients at risk of developing ARDS

Company plans to study lenzilumab in COVID-19 patients to prevent ARDS and mortality with CTI Clinical Trial and Consulting Services (CTI)

COVID-19 is an infectious disease caused by SARS-CoV-2. COVID-19 has become a global pandemic, with over 460,000 confirmed cases and over 21,000 deaths reported to date. Patients with severe cases of COVID-19 experience severe viral pneumonia that can progress to acute respiratory distress syndrome (ARDS) and death.

ARDS is an acute, life-threatening inflammatory lung injury characterized by hypoxia a lack of oxygen to the tissue and stiff lungs due to increased pulmonary vascular permeability. ARDS necessitates hospitalization and mechanical ventilation. A rapid increase in patients with ARDS presents a major challenge for the global public health system given limited hospital beds and ventilators. When implementing standard of care, including mechanical ventilation, ARDS has an overall mortality rate of greater than 40 percent.

Humanigen, Inc. study is focused on the belief that its GM-CSF neutralization and gene-editing platform technologies have the potential to reduce the inflammatory cascade associated with coronavirus infection as well as the serious and potentially life-threatening CAR-T therapy-related side effects while preserving and potentially improving the efficacy of the CAR-T therapy itself, thus breaking the efficacy/toxicity linkage.

The companys immediate focus is to prevent or minimize the cytokine storm that precedes severe lung dysfunction and ARDS in cases of pneumonia-associated SARS-CoV-2 infection and also in combining FDA-approved and development stage CAR-T therapies with lenzilumab, the companys proprietary Humaneered anti-human-GM-CSF immunotherapy, which is its lead product candidate.

CTI

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Covington-based CTI partners with Humanigen for study of Lenzilumab for treatment of Coronavirus - User-generated content

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WALTHAM, Mass., March 31, 2020 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies and a mission to develop transformative medicines for devastating diseases, today announced it has closed an oversubscribed $60 million Series A financing. Seed venture investors F-Prime Capital and New Enterprise Associates (NEA) co-led the round alongside new investor Atlas Venture, with participation from seed investors Alexandria Venture Investments, Lonza and Partners Innovation Fund.

The proceeds will be used to advance the companys platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.

The company is led by recently appointed Chief Executive Officer Rick Modi, who has a proven track record of building value at companies including AveXis, InterMune, MedImmune and Centocor. Joining the board of directors as part of the financing are Dave Grayzel, M.D., Partner, Atlas Venture; Ed Mathers, General Partner, NEA; and Robert Weisskoff, Ph.D., Partner, F-Prime Capital. Industry veteran and gene therapy leader Sean Nolan will chair the board.

At Affinia Therapeutics, were setting a new standard in gene therapies by leveraging our proprietary platform to methodically engineer novel AAV vectors and gene therapies that have remarkable targeting properties, said Modi, CEO of Affinia. We are pleased to partner with such a distinguished syndicate to advance our platform and investigational product candidates toward the clinic for patients in need.

The companys technology was licensed from Lonza and Massachusetts Eye and Ear. It was developed at the Grousbeck Gene Therapy Center and further advanced under a sponsored research agreement with Lonza led by Luk Vandenberghe, Ph.D., Associate Professor at Mass. Eye and Ear and Harvard Medical School and a co-inventor of AAV9.

In addition to Vandenberghe, joining Affinia Therapeutics as scientific co-founders are Botond Roska, M.D., Ph.D., Director, Institute of Molecular and Clinical Ophthalmology Basel; Aaron Tward, M.D., Ph.D., Assistant Professor, University of California, San Francisco; and Eric Zinn, Ph.D. student, Mass. Eye and Ear and Harvard University. Together, these scientists have authored more than 200 papers and filed more than 20 patents in the field of gene therapy.

Affinia Therapeutics is bringing together complementary expertise allowing us to realize a rational design future for AAV vectors, promoters and other components of gene therapies. By leveraging synthetic and systems biology combined with high-throughput screening and tissue and single-cell resolution, we are aspiring to achieve much-needed improved pharmacological control of this novel modality in medicine, said Vandenberghe, Director, Grousbeck Gene Therapy Center at Mass. Eye and Ear.

The Series A financing comes after Vandenberghe and his team successfully developed AAVSmartLibraries comprising thousands of functional novel AAV vectors. Each vector is uniquely identified, and the libraries can be screened across species for parameters of high interest, including tissue tropism, manufacturing yield and pre-existing immunity. Observations arising from each library screen provide insights into the vectors structure-function, enabling the rational design of novel vectors and gene therapies with remarkably improved properties. Affinia Therapeutics has potentially the worlds largest library of patented functional AAV vectors.

Affinia Therapeutics methodical process for designing and evaluating vectors is a differentiated approach to gene therapy, and the highly experienced leadership team will help carry these discoveries to the development, manufacturing and commercialization of transformative medicines, said Mathers, General Partner at NEA. We are pleased to accelerate the impact of this exciting field.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need.www.affiniatx.com.

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Affinia Therapeutics Raises $60M in Series A Financing to Advance Rational Design AAV Platform and Transformative Gene Therapies - GlobeNewswire

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Demonstrates broad biodistribution, including across the blood-brain barrier into the central nervous system, and into skeletal muscle, in non-human primates (NHPs) after systemic administration

Durable and therapeutically relevant drug concentrations achieved in NHPs after single intravenous dose

Potent cell-based activity and allele-specific enrichment in patient-derived cell lines

Platform validation data supports expansion of the therapeutic pipeline into new organ systems previously unreachable with first-generation antisense oligonucleotide technology

Management to hold a conference call today at 8 a.m. ET

PITTSBURGH, March 31, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq:NBSE) (NeuBase or the Company), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies to address genetic diseases, today announced positive preclinical data from its pharmacokinetics studies in non-human primates (NHPs) and in vitro pharmacodynamics data in patient-derived cell lines. NeuBase believes these data validate the key advantages of the proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform and support the Companys decision to advance the development of its Huntingtons disease (HD) and myotonic dystrophy type 1 (DM1) programs, as well as the potential expansion of its therapeutic pipeline into other indications.

Dr. George Church, professor of genetics at Harvard Medical School and member of the National Academy of Sciences, stated, Given the activity and broad biodistribution observed in these studies and the potential for easier target definition, I believe the PATrOL technology may have a potent impact on the future of drug development and treatment of genetic diseases.

Non-Human Primate Pharmacokinetic Study

Quantitative whole-body autoradiography was performed on NHPs.A PATrOL-enabled compound was radio-labeled, and theresulting material was injected into NHPs at 5 mg/kg via a bolus tail vein injection. At four hours, twelve hours, and seven days post-dosing, NHPs were sacrificed andsectioned into 40 m slices.Slices were exposed to autoradiography imaging plates alongside a dilution series of radioactive PNA in whole blood.Upon imaging, the dilution series enabled an analysis of the amount of compound in each of the tissues. In addition, prior to sacrifice, whole blood, urine, and feces were collected from the NHPs at specified timepoints.The major conclusions from this study include:

Rapid uptake of compound out of the bodys circulation after systemic intravenous administration, with a half-life in circulation of approximately 1.5 hours;

Compound penetrates every organ system studied, including the central nervous system and skeletal muscle;

Compound crosses the blood-brain barrier and into the key deep brain structures, including the caudate, supporting a key capability for the development of the Companys lead program in HD; Delivery of the compound to skeletal muscle, the primary organ system that is affected in DM1;Because both HD and DM1 have manifestations outside of the primary affected organ, the broad biodistribution of the compounds may enable a potential whole-body therapeutic solution in both indications.

96% of administered compound remained in vivo after a one-week period (latest timepoint tested);Redistribution over one week after administration between organ systems enriches concentrations in key brain regions up to two-fold, including in those deep brain structures most relevant for HD;Retention of ~90% of compound concentrations achieved in skeletal muscle over the course of one-week post-single-dose administration; and

Patient-Derived Huntingtons Cell Line Pharmacodynamic Studies

Multiple Huntingtons disease candidate compounds were incubated with HD-derived cells and assayed for their toxicity and their ability to selectively knock down mutant huntingtin protein (mHTT) expression by engaging with the CAG repeat expansion in the huntingtin (HTT) gene transcript. Multi-well plates were seeded with cells and candidates were added to the culture at various concentrations.Cells were grown for three days and thereafter assayed for cell death.Cell pellets were also collected, lysed, and run on gradient SDS-PAGE gels.Following the transfer of the proteins to a membrane, the membrane was probed with anti-huntingtin and anti-beta-actin antibodies.Secondary antibodies were used to image the immunoblots.The beta-actin bands were used to normalize the amount of protein across the wells.The amounts of mutant and wild type huntingtin protein in treated cells were compared to untreated cells to determine the level of knockdown.The major conclusions from this study include:

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Activity in engaging target disease-causing transcripts and knocking-down resultant malfunctioning mHTT protein levels preferentially over normal HTT protein knock-down; and

Dose limiting toxicities were not observed relative to a control either at or above the doses demonstrating activity in human cells in vitro.

In addition, PATrOL enabled compounds were generally well-tolerated in vivo after systemic administration, both after single dose administration in NHPs and multi dose administration in mice for over a month.

We believe the PATrOL platform has the potential to create drugs that are easy for patients to take at infrequent intervals after they have tested positive for a genetic disease but before symptoms emerge, said Dietrich Stephan, Ph.D., chief executive officer of NeuBase. We believe the best way to effectively manage degenerative genetic diseases is to get ahead of the disease process, and we believe that can only be achieved with early diagnosis coupled with well-tolerated, effective, and easily administered therapies.

Dr. Robert Friedlander, chief medical officer of NeuBase and member of the National Academy of Medicine, stated, An allele specific approach that can be systemically administered and cross the blood brain barrier would be an ideal drug profile for many untreatablegenetic diseases.I believe that NeuBase is moving towards realizing this goal.

The intersection of the NHP pharmacokinetic data and the in vitro patient-derived pharmacodynamic data provides a roadmap to create a pipeline of therapeutic candidates which can reach target tissues of interest after systemic administration and achieve the desired activity at that dose. NeuBase believes the data from these studies support the advancement of the Companys HD and DM1 programs into lead optimization and subsequent IND-enabling studies, as well as provide a roadmap for the future expansion of the Companys therapeutic pipeline into other indications, including oncology.

Dr. Sam Broder, former Director of the National Cancer Institute of the National Institutes of Health and member of the National Academy of Sciences, stated, I believe that the NeuBase strategy of targeting transcripts before they become dangerous mutant proteins has the potential to deliver a dramatic improvement in our collective capabilities to effectively treat a wide range of genetic diseases, including some of the most deadly cancers, by targeting driver mutations and accelerating immunotherapy capabilities.

Conference Call

NeuBase Therapeutics, Inc. will discuss these data and next steps for development during a webcasted conference call with slides today, March 31, 2020, at 8:00 a.m. ET. The live and archived webcast of this presentation can be accessed through the IR Calendar page on the Investors section of the Companys website, http://www.neubasetherapeutics.com. The dial-in details for the call are 877-451-6152 (domestic) or +1-201-389-0879 (international), and conference ID: 13701118. The archived webcasts will be available for approximately 30 days following the presentation date.

About NeuBase Therapeutics

NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform is designed to permit the rapid development of targeted drugs, thereby potentially increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements include, among other things, statements regarding the Companys goals and plans and the Companys pharmacokinetics and pharmacodynamics studies. These forward-looking statements are distinguished by use of words such as will, would, anticipate, expect, believe, designed, plan, or intend, the negative of these terms, and similar references to future periods. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those described in the risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Companys current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Companys plans to develop and commercialize its product candidates; the Companys plans to commence clinical trials in Huntingtons disease and myotonic dystrophy type 1 and to potentially expand the pipeline into other indications; the utility of the preclinical data generated in existing studies performed by the Company in determining the results of potential future clinical trials and of the potential benefits of the PATrOL platform technology; the timing of initiation of the Companys planned clinical trials; the timing of the availability of data from the Companys clinical trials; the timing of any planned investigational new drug application or new drug application; the Companys plans to research, develop and commercialize its current and potential future product candidates; the clinical utility, potential benefits and market acceptance of the Companys current and potential future product candidates; the Companys commercialization, marketing and manufacturing capabilities and strategy; the Companys ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCDaniel@lifesciadvisors.comOP: (617) 535-7746

NeuBase Media Contact:Travis Kruse, Ph.D.Russo Partners, LLCtravis.kruse@russopartnersllc.comOP: (212) 845-4272

View post:
NeuBase Therapeutics Announces Positive, Preclinical Data Validating its Novel Genetic Therapy PATrOL Platform - Yahoo Finance

Recommendation and review posted by Bethany Smith

Gene Therapy Market industry development trends and strategic plan for positive Growth discuss in research report.The global gene therapy market is estimated to be over US$ 411.2 Mn in 2018. It is anticipated to grow at a double-digit CAGR of 33.1% from 2019 to 2030.

This press release was orginally distributed by SBWire

New York, NY -- (SBWIRE) -- 03/31/2020 -- As per research findings of Market Industry Reports (MIR), increasing number of product approvals & R&D activities, supportive government initiatives in the gene therapy arena are set to open new avenues for stakeholders.

Major Players Include:

The global gene therapy market is largely governed by prominent players such as Novartis AG, UniQure N.V., Regenxbio Inc., Sibiono GeneTech Co. Ltd., Amgen Inc., Voyager Therapeutics, Sarepta Therapeutics, Inc., Spark Therapeutics, Inc., Audentes Therapeutics, and Orchard Therapeutics plc, among others.

Get Access to Sample Pages @ https://www.marketindustryreports.com/pdf/149

Growth Factors:

Growth opportunities in the global gene therapy market looks promising during the forecast period. Rising incidences of chronic diseases as well as genetic disorders globally, favorable initiatives from governments to support research & development of gene therapy products and technological advancements are some of the key factors driving the global gene therapy market.

Similarly, increased focus of players in this market for getting approvals for gene therapy products is anticipated to boost the gene therapy market significantly during the forecast period. For instance, in May 2019, AveXis, a U.S.-based company received FDA's approval for Zolgensma. Zolgensma is used for the treatment of a rare genetic disorder and Spinal Muscular Atrophy (SMA) in pediatric patients below the age of 2 years."Companies are striving to discover, develop, and deliver gene therapies for rare genetic disorders,"

From a regional perspective, Europe is expected to witness the highest CAGR in the coming years due to presence of several gene therapy products in the development stage. Whereas, North America accounts for the largest share in the gene therapy market. The large share of this region can be attributed to rising incidences of chronic diseases & genetic disorders such as cancer, hemophilia, Multiple Sclerosis (MS), and others.

For deep-rooted insights on this topic, Go for Interesting Discount @ https://www.marketindustryreports.com/discount/149

MARKET ECOSYSTEM/CLASSIFICATION

By Vector Type

Viral VectorsAdenovirusLentivirusRetrovirusAdeno-associated virusHerpes simplex virusPoxvirusVaccinia virusOthersNon-viral VectorsNaked plasmid vectorGene gunElectroporationLipofectionOthers

By Gene TypeAntigenCytokineTumor SuppressorSuicideDeficiencyGrowth FactorsReceptorsOthers

By ApplicationOncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious DiseasesOther Diseases

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Deep-rooted quantitative and qualitative insights at segment & sub-segment levelGlobal insights with country levels informationComprehensive insights on market dynamics, and business environment Insights on 'Key Strategies' followed by leading players of the marketAccess to 'Latest Strategic Developments' registered by leading players of the market

CUSTOMIZATION OPTIONS:

Key Distributors ListFree addition of 'Company Profiles'Clientele Base Assessment

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About Market Industry ReportsMarket Industry Reports is a global leader in market measurement and advisory services. It is a 100% subsidiary of ExlTech. The firm has always been at the forefront of innovations to address worldwide industry trends and opportunities. We offer our clients a unique depth of market intelligence in an actionable format to move their business forward. Our analysis incorporates consumer study in more than 100 countries, providing a tactical approach to drive sustained business growth. We continue to pioneer state-of-the-art approach in research & analysis that will help you to overcome complexities and stay ahead of the curve. By nurturing the perception of genius and optimized market intelligence, we unfold contingencies for our clients in the evolving world of technology, mega-trends, and industry convergence. We inspire our clients to empower and shape their businesses and to build world-class products.

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For more information on this press release visit: http://www.sbwire.com/press-releases/gene-therapy-market-companies-outlook-and-trends-likely-to-witness-cagr-of-331-from-2019-to-2030-1285124.htm

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Gene Therapy Market Companies Outlook and Trends Likely to Witness CAGR of 33.1% from 2019 to 2030 - Press Release - Digital Journal

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Hemophilia Treatment Market to 2027 - Global Analysis and Forecasts by Product; Disease; Treatment Type; Therapy; End User, and Geography" report has been added to ResearchAndMarkets.com's offering.

The global hemophilia treatment is expected to reach US$ 44,089.71 million in 2027 from US$ 14,454.81 Mn in 2019. The market is estimated to grow with a CAGR of 15.9% from 2020-2027.

The growth of the hemophilia treatment is primarily attributed to the factors such as increasing prevalence of hemophilia and rise in favorable government initiatives leading to launch of new products in the market. However, high cost related with hemophilia treatment is likely to pose a negative impact on the market growth. On the other hand, growth opportunities in emerging regions are likely to have a positive impact on the growth of the global hemophilia treatment during the forecast period.

According to data from the National Hemophilia Foundation in 2020, Hemophilia A happens in 1 in 5,000 live male births. Hemophilia A is about four times as common as hemophilia B. The number of people with hemophilia in the United States is anticipated to be around 20,000 individuals. Moreover, the incidence of hemophilia is not known across the globe but projected at above 400,000 people. Almost 75% of people with hemophilia worldwide are either undiagnosed or receive disparate treatment.

In Italy, according to the data of the National Center for Biotechnology Information (NCBI) in 2017, the number of registered people with bleeding disorders increased from about 7000 in 2000 to around 8500 in 2011 and more than 11,000 in 2015. The trend is due to an upsurge in the number of patients who are recorded, mainly in those with vWD type 1, mild hemophilia, or other factor deficiencies. Thus, increasing prevalence of hemophilia, high cases of under-diagnosed hemophilia cases, and improving healthcare infrastructure will eventually grow the market for hemophilia treatment in the forecast period.

Global hemophilia treatment, based on the product, is segmented into plasma derived coagulation factor concentrates plasma derived coagulation factor concentrates, desmopressin, antifibrinolytic agents. In 2019, the recombinant coagulation factor concentrates held the largest share of the market, by product. Also, recombinant coagulation factor concentrates segment is expected to grow at the fastest rate during the coming years owing to increasing adoption of integrated systems across the globe.

Reasons to Buy

Key Topics Covered

1. Introduction

1.1 Scope of the Study

1.2 Report Guidance

1.3 Market Segmentation

1.3.1 Global Hemophilia Treatment Market - By Product

1.3.2 Global Hemophilia Treatment Market - By Disease

1.3.3 Global Hemophilia Treatment Market - By Treatment Type

1.3.4 Global Hemophilia Treatment Market - By Therapy

1.3.5 Global Hemophilia Treatment Market - By End User

1.3.6 Global Hemophilia Treatment Market - By Geography

2. Global Hemophilia Treatment Market - Key Takeaways

3. Research Methodology

3.1 Coverage

3.2 Secondary Research

3.3 Primary Research

4. Hemophilia Treatment- Market Landscape

4.1 Overview

4.2 PEST Analysis

4.2.1 North America - PEST Analysis

4.2.2 Europe: PEST Analysis

4.2.3 APAC: PEST Analysis

4.2.4 MEA: PEST Analysis

4.2.5 South & Central America - PEST Analysis

4.3 Expert Opinion

5. Hemophilia Treatment Market - Key Market Dynamics

5.1 Market Drivers

5.1.1 Increasing Prevalence of Hemophilia

5.1.2 Rise in Favorable Government Initiatives

5.2 Market Restraints

5.2.1 High Cost Related with Hemophilia Treatment

5.3 Market Opportunities

5.3.1 Growth in the Global Healthcare Market

5.4 Future Trends

5.4.1 Research Activities and Product Innovations

5.5 Impact Analysis

6. Hemophilia Treatment Market - Global Analysis

6.1 Global Hemophilia Treatment Market Revenue Forecast And Analysis

6.2 Global Hemophilia Treatment Market, By Geography - Forecast And Analysis

6.3 Positioning of Key Players

6.3.1 Bayer AG

6.3.2 Pfizer Inc.

7. Global Hemophilia Treatment Market Analysis- by Product

7.1 Overview

7.2 Hemophilia Treatment Market Revenue Share, by Product (2019 and 2027)

7.3 Plasma-Derived Coagulation Factor Concentrates

7.4 Recombinant Coagulation Factor Concentrates

7.5 Desmopressin

7.6 Antifibrinolytic Agents

8. Hemophilia Treatment Market Analysis - by Disease

8.1 Overview

8.2 Hemophilia Treatment Market, by Disease 2019 and 2027 (%)

8.3 Hemophilia A

8.4 Hemophilia B

8.5 Hemophilia C

9. Hemophilia Treatment Market Analysis - by Treatment Type

9.1 Overview

9.2 Hemophilia Treatment Market Revenue Share, by Treatment Type (2019 and 2027)

9.3 Prophylaxis

9.4 On-demand

10. Hemophilia Treatment Market Analysis - by Therapy

10.1 Overview

10.2 Hemophilia Treatment Market, by Therapy 2019 and 2027 (%)

10.3 Replacement Therapy

10.4 ITI Therapy

10.5 Gene Therapy

11. Hemophilia Treatment Market Analysis - by End User

11.1 Overview

11.2 Hemophilia Treatment Market, by End User 2019 and 2027 (%)

11.3 Hospitals & Clinics

11.4 Hemophilia Treatment Centers

11.5 Ambulatory Surgical Centers

12. Hemophilia Treatment Market - Geographic Analysis

12.1 North America: Hemophilia Treatment Market

12.2 Europe: Hemophilia Treatment Market

12.3 APAC: Hemophilia Treatment Market

12.4 Middle East & Africa: Hemophilia Treatment Market

12.5 South And Central America: Hemophilia Treatment Market

13. Global Hemophilia Treatment Market - Industry Landscape

13.1 Overview

13.2 Growth Strategies Done By the Companies in the Market, (%)

13.3 Organic Developments Done By The Companies in The Market

13.4 Inorganic Developments Done by The Companies In The Market

14. Company Profiles

14.1 Bayer AG

14.2 Sanofi

14.3 F. Hoffmann-La Roche Ltd.

14.4 Kedrion S.p.A.

14.5 CSL Limited

14.6 Biotest AG.

14.7 Pfizer Inc.

14.8 Novo Nordisk A/S

14.9 Octapharma AG

Continued here:
Hemophilia Treatment Market Study & Forecast to 2027 by Product, Disease, Treatment Type, Therapy, End-user and Geography - ResearchAndMarkets.com...

Recommendation and review posted by Bethany Smith

A new research study titled Global Hemophilia Gene Therapy Market Growth (Status and Outlook) 2020-2025 has been presented by MRInsights.biz offering a comprehensive analysis of the market through which users can benefit from the complete market research report with all the required useful information about this global Hemophilia Gene Therapy market. The report covers the latest and upcoming industry trends and offers a global spectrum of the market and future forecast from 2020 to 2025 years. The report offers a detailed examination of opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, and developments.

The market is bifurcated into product type, application, key manufacturers and key regions and countries. The report assists users to achieve competitive leverage with acquiring and preserving market position in the global Hemophilia Gene Therapy industry. The report provides an investigation into the global market status, shares, supply-demand, market drivers, challenges and opportunities, and geological areas. The report then serves information on sales and market share estimates by-product as well as a profile of the companys business. The study emphasizes on consumption, market share and growth rate of the industry. The research helps readers to understand strategies to make sound investments.

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On the basis of types, the market is primarily split into: Hemophilia A, Hemophilia B

On the basis of applications, the market covers: Hemophilia A Gene Therapy, Hemophilia B Gene Therapy

Additionally company basic information, manufacturing base, and competitors list is being provided for each listed manufacturers: Spark Therapeutics, Ultragenyx, Sangamo Therapeutics, Bioverativ, Shire PLC, Freeline Therapeutics, BioMarin, uniQure

All key regions and countries are assessed here on the basis of company, type of product, and application covering Americas (United States, Canada, Mexico, Brazil), APAC (China, Japan, Korea, Southeast Asia, India, Australia), Europe (Germany, France, UK, Italy, Russia, Spain), Middle East & Africa (Egypt, South Africa, Israel, Turkey, GCC Countries)

The study presents in-depth insights into each of the leading end-user verticals along with annual forecasts to 2025. The report includes global Hemophilia Gene Therapy market consumption analysis by application as well as analysis of value, product utility, market percentage, and production market share by type. Key attributes of the leading companies are displayed that includes company overview, financial overview, recent developments, development strategies, and SWOTs.

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The Report Addresses The Following Queries Related To The Market:

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

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Global Hemophilia Gene Therapy Market 2020 Industry Size, Development Status, Estimated CAGR, and Forecast to 2025 - Galus Australis

Recommendation and review posted by Bethany Smith

A leading market research firmFacts & Factors (FnF)added a research report onGene Therapy Market By Type (Germ Line Gene Therapy and Somatic Gene Therapy), By Vector Type (Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome), and By Therapy Area (Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027includes 190+ pages research report with TOC included in its research database. The research report portraying a wide-ranging assessment of theGene Therapy marketincludes the rate of expansion of the Gene Therapy market over the predicted duration. Providing a succinct outline, it concludes the valuation and size of the Gene Therapy market in the near future. Additionally, it also comprises the foremost contributing aspects to the development of the Gene Therapy market as well as the leading players in the market along with their market share. The top outstanding industry players/manufacturers are also included in this report to understand the companys business strategies, sales, and factor of growth.

The Gene Therapy market research report offers valuable insights pertaining to the revenue segmentation, business summary, and product offerings of the foremost market players. The study also estimates the expansion of the well-known market players through SWOT study. Also, it considers the most recent developments in the market whilst estimating the growth of major market players. Furthermore, in the Gene Therapy market size report, the main product category and segments along with its sub-segments of the Keyword market are elaborated.

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This free report sample includes: The report covers present status and future prospects. The report analyses market trends, size, and forecast in different geographically. The report provides market competition overview among the Top companies. The report provides a complete analysis of the current and emerging market trends and opportunities. Example pages from the report. FnF research methodology.

The Gene Therapy market research report also states the latest floats in the Gene Therapy market and numerous opportunities for the growth of the Gene Therapy market in the upcoming period. The study utilizes many methodological tools to estimate market expansion in the predicted period.

Thesize of Gene Therapy marketis divided based on the product type, purchaser, and application segments. The industry growth of each segment is assessed along with the prediction of their growth in the near future. The relevant data and statistics gathered from the regulatory authorities have been portrayed in the report to assess the development of segments. Further, the trends in the Gene Therapy market are also branched out on the basis of regions such as the Middle East & Africa, Asia Pacific, Latin America, North America, and Europe.

To Know What is Size, Share of the Gene Therapy Market, Ask a Free Sample Report:https://www.fnfresearch.com/sample/gene-therapy-market-by-type-germ-line-gene

Major Company Profiles Covered in This Report:

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The Gene Therapy market analysis research report is drafted on the basis of a brief assessment and huge data collected from the Gene Therapy market. The data collected include current industry trends and requirements associated with services & manufacturing goods.

The report on Gene Therapy market worth segments and defines the Gene Therapy market considering the in-detailed forecasts and study of the market size. The report further includes various figures, data tables, and in-depth TOC on the Gene Therapy market.

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An in-depth analysis of the most recent developments and novel technological advancements provides the users with a free hand to introduce their exceptional products and processes to update the service contribution. The global share of Gene Therapy market report emphasizes the most recent trends, growth, and new business opportunities to provide a comprehensive review of the global market. The demand proportion and development of innovative technologies also are revealed in the Gene Therapy market report.

The statistics in the data collected are graphically presented in theGene Therapy market size and trendsresearch report. It also includes key performers, vendors, and suppliers. The report highlights the command and drivers influencing the market.

What Gene Therapy Market research report offers?

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Facts & Factorsis a leading market research company and offers customized research reports and consulting services. Facts & Factors aims at management consulting, industry chain research, and advanced research to assist our clients by providing planned revenue model for their business. Our report and services are used by prestigious academic institutions, start-ups, and companies globally to understand the international and regional business background. Our wide-ranging database offers statistics and detailed analysis of different industries worldwide that help the clients in achieving sustainable progress. The well-organized reports help clients in developing strategies and making informed business decisions.

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Global Gene Therapy Market to Reflect Impressive Growth Rate by 2026 - Global Industry News 24

Recommendation and review posted by Bethany Smith

Report Description

A recent market intelligence report that is published by Data Insights Partner onGene Therapy marketmakes an offering of in-depth analysis of segments and sub-segments in the regional and international Gene Therapy market. The research also emphasizes on the impact of restraints, drivers, and macro indicators on the regional and world Gene Therapy market over the short as well as long period of time. A detailed presentation of forecast, trends, and dollar values of international Gene Therapy market is offered. In accordance with the report, the global Gene Therapy market is projected to expand at a CAGR of 30% over the period of forecast.

Market Insight, Drivers, Restraints& Opportunity of the Market:

Gene therapy is a medical procedure which replaces defective genes or introduces new genes n order to prevent or cure genetic disorders. This procedure has become a bench mark in medical industry as there is no requirement of surgery or drugs or other procedure which has side effects on the individuals. Gene therapy was first commercialized in China in 2004 by China based SiBono Gene Tech (product Gendicine).

The global gene therapy market has been expanding due to the rigorous research conducted in the field of genetics. The rising awareness about the capability of cure of several rare genetic diseases by gene therapy is another important driver which leads the global gene therapy market during the forecast period. Gene therapy has capability cure several life threatening diseases such as cancer, cardiac diseases, AIDS, cystic fibrosis, age-related disorders, sickle cell anemia etc. In March 2019, the director of the National Health Institute (NIH), the U.S. announced that the recent clinical trials on the gene therapy for the treatment of sickle cell anemia showed promising result- therefore, increasing prevalence of aforementioned lie threatening diseases would likely to drive the growth of the global gene therapy market during the forecast period.

Request for Report Sample:https://datainsightspartner.com/request-for-sample?ref=8

On the other hand, treatment cost and stringent regulatory conditions etc. may hamper the growth of the global gene therapy market in the upcoming future. The results of Gendicine clinical trials were published in 2003 and the medicine got approval by the China State Food and Drug Administration in the same year. Although approved in China, Gendicines use is not very promising outside China. There are several concerns among the researchers about the quality of the clinical trials performed and safety and efficacy of the treatment. However, Gendicines equivalent Advexin (company Introgen Therapueitcs) is still waiting for the FDA approval.

Increasing investment to the gene therapy related research (around 10 Bn was invested in 2015 by private and public organizations), new product developments such as (Zolgensma in 2019), strategic alliance among the key players (such as collaboration between Axovant and Yposkesi) would bring the global gene therapy market an opportunity to propel during the forecast period. In May 2019, Avexis (a Novartis company) has got the FDA approval for Zolgensma for treatment of spinal muscular atrophy for the pediatric patients (less than 2 years of age).

Segment Covered:

This market intelligence report on the global gene therapy market encompasses market segments based on product, application, target user and geography. On the basis of product, the sub-markets is segmented into Yescarta, Kymriah, Strimvelis, Gendicine, Zolgensma and others (Advexin). Based on application, the global gene therapy market has been segregated into large B-Cell lymphoma, Car T Cell therapy, ADA-SCID (adenosine deaminase deficiency), muscular atrophy, head and neck squamus cell carcinoma, others (Crigler-Najjar syndrome). By target user, the global gene therapy market is also classified into adult and pediatric. By Geography, the global gene therapy market has been divided into North America (the U.S., Canada), Latin America (Brazil, Mexico, Argentina and other countries), Europe (Germany, France, the U.K., Spain, Italy, Russia, and other countries), Asia Pacific (India, Japan, China, Australia and New Zealand and other countries), Middle East and Africa (GCC, South Africa, Israel and Other countries).

Profiling of Market Players:

This business intelligence report offers profiling of reputed companies that are operating in the market. Companies such as Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others have been profiled into detail so as to offer a glimpse of the market leaders. Moreover, parameters such as gene therapy market related investment & spending and developments by major players of the market are tracked in this global report.

Report Highlights:

In-depth analysis of the micro and macro indicators, market trends, and forecasts of demand is offered by this business intelligence report. Furthermore, the report offers a vivid picture of the factors that are steering and restraining the growth of this market across all geographical segments. In addition to that, IGR-Growth Matrix analysis is also provided in the report so as to share insight of the investment areas that new or existing market players can take into consideration. Various analytical tools such as DRO analysis, Porters five forces analysis has been used in this report to present a clear picture of the market. The study focuses on the present market trends and provides market forecast from the year 2017-2027. Emerging trends that would shape the market demand in the years to come have been highlighted in this report. A competitive analysis in each of the geographical segments gives an insight into market share of the global players.

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Salient Features:

This study offers comprehensive yet detailed analysis of the Gene Therapy market, size of the market (US$ Mn), and Compound Annual Growth Rate (CAGR (%)) for the period of forecast: 2019 2027, taking into account 2017 as the base year

It explains upcoming revenue opportunities across various market segments and attractive matrix of investment proposition for the said market

This market intelligence report also offers pivotal insights about various market opportunities, restraints, drivers, launch of new products, competitive market strategies of leading market players, emerging market trends, and regional outlook

Profiling of key market players in the world Gene Therapy market is done by taking into account various parameters such as company strategies, distribution strategies, product portfolio, financial performance, key developments, geographical presence, and company overview

Leading market players covered this report comprise names such as. Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others

The data of this report would allow management authorities and marketers of companies alike to take informed decision when it comes to launch of products, government initiatives, marketing tactics and expansion, and technical up gradation

The world market for Gene Therapy market caters to the needs of various stakeholders pertaining to this industry, namely suppliers, manufacturers, investors, and distributors for Gene Therapy market. The research also caters to the rising needs of consulting and research firms, financial analysts, and new market entrants

Research methodologies that have been adopted for the purpose of this study have been clearly elaborated so as to facilitate better understanding of the reports

Reports have been made based on the guidelines as mandated by General Data Protection Regulation

Ample number of examples and case studies have been taken into consideration before coming to a conclusion

Reasons to buy:

vIdentify opportunities and plan strategies by having a strong understanding of the investment opportunities in the Gene Therapy market

vIdentification of key factors driving investment opportunities in the Gene Therapy market

vFacilitate decision-making based on strong historic and forecast data

vPosition yourself to gain the maximum advantage of the industrys growth potential

vDevelop strategies based on the latest regulatory events

vIdentify key partners and business development avenues

vRespond to your competitors business structure, strategy and prospects

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Gene Therapy Market Regulations and Competitive Landscape Outlook size 2027 - Skyline Gazette

Recommendation and review posted by Bethany Smith

The latest report on the Global Gene Therapy Technologies Market Report 2020-2026 is a systematic and insightful compilation of valuable evaluations of Gene Therapy Technologies market and relevant aspects. The report offers an in depth exploration of the market and its scope, trends, structure, production, profitability and maturity. The precise evaluation of market size, share, revenue, sales volume, demand, and rate of growth involved within the report drive investors, industry experts, researchers, also as novice and well-established market players to grasp the general Gene Therapy Technologies market structure.

NOTE: Our reports include the analysis of the impact of COVID-19 on this industry. Our new sample is updated which correspond in new report showing impact of Covid-19 on Industry trends. Also we are offering 20% discount

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The Gene Therapy Technologies market report also delivers an in-depth analysis of the emerging industry trends along side the restraints, drivers, and opportunities within the Gene Therapy Technologies market to supply worthwhile insights also as a gift scenario for generating right decision. Moreover, the new report on the Gene Therapy Technologies industry covers the prominent vendors within the universal market alongside SWOT analysis, fiscal overview and major developments.

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Leading companies reviewed in the Gene Therapy Technologies report are:

Bluebird bioAdaptimmuneGlaxoSmithKlineMerckCelgeneShanghai Sunway BiotechBioCancellShenzhen SiBiono GeneTechSynerGene TherapeuticsOncoGenex PharmaceuticalsGenelux CorporationCell GenesysAdvantageneGenVecBioCancellCelgeneEpeius BiotechnologiesIntrogen TherapeuticsZiopharm OncologyShenzhen SiBiono GeneTechAltor Bioscience

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The product segment of the report offers product market information such as demand, supply and market value of the product.

The application of product in terms of USD value is represented in numerical and graphical format for all the major regional markets.The Gene Therapy Technologies market report is segmented into Type by following categories;Type IType II

The Gene Therapy Technologies market report is segmented into Application by following categories;Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovasular DiseasesOthers

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Global Gene Therapy Technologies Market Survey 2020-2026 Growth Prediction by Manufacturers Bluebird bio, Adaptimmune, GlaxoSmithKline, Merck - Galus...

Recommendation and review posted by Bethany Smith

Yuri Molchan

Major crypto platform founders support anti-aging research, saying that a bigger population could live even with finite resources

Ethereums Vitalik Buterin, IOTAs David Snsteb and Trons Justin Sun are discussing anti-aging research andsupporting it. Buterin admittedthat thisidea is weird for many peoplethese days, though, he also calledaging a humanitarian disaster.

In the course of a recent discussion of weird ideas on Twitter, the co-founder of Ethereum, Vitalik Buterin, suggested discussing anti-aging research, calling aging a humanitarian disaster.

He stated that every two years, as many people die from aging as didin World War 2. Additionally, he saidthat theissue burdens social systems and families. Buterin hopes that soon scientists will be able to significantly extend the human life span.

When asked about the planet getting overpopulated and the issue of the resources coming to an end, the Ethereum co-founder stated:

On average a new person contributes more to the resources available to existing humans than they deplete. (This is due to their productive labor, scientific research, the change they become the next Norman Borlaug, etc...)

We are nowhere near any theoretical bounds on our ability to use those resources. Our efficiency in using limited resources has increased by orders of magnitude in the last two centuries, and can rise orders of magnitude more.

Must Read

IOTA founder, David Snsteb, has responded, saying that in 2020 the discussion on life-extension measures should not at all be considered a weird idea, unlike it was fifteen years ago, in 2005.

Now, Snsteb tweeted, this should be the universal common sense position.

The Tron founder Justin Sun briefly joinedthe discussion.

In an exclusive interview with U.Today, the IOHK CEO and Cardano founder Charles Hoskinson also shared his take on the subject of anti-aging.

While describing possible trends of the future, he mentioned a largely growing market of life extension products and services. He recalled thewell-known example of a jellyfish that can regenerate and thus live forever.

So there's probably going to be a considerable amount of improvement in regenerative biology and an improvement in a human lifespan extension and new therapies and medicines to keep us healthy into our deep old age. So then what does that mean? It means you have a human race that doesn't recycle every 80 years. They may last a lot longer.

He also suggestedthat as the anti-aging trend likely gets wider and stronger, there will be fewer religious people accepting death as a natural part of human life.

See the article here:
Vitalik Buterin Wants to Rid Humanity of Aging, David Snsteb and Justin Sun Join In - U.Today

Recommendation and review posted by Bethany Smith

Great Panther Mining (GPL +0.4%) says that it aims to ramp up exploration, expand its resources and improve its finances

GPL said that in 2020, the firm would invest a minimum of $11M aimed at realizing exploration potential at its assets, with particular focus on mine life extension at the Brazil mine and a possible re-start of the Guanajuato Mine

Q4 revenue came in at $65.6M, +381% Y/Y, reflecting the acquisition of Tucano.

Net loss was $28.1M, as mine operating earnings were offset by an impairment of Tucano goodwill.

Great Panther said it could not yet provide company-wide guidance for 2020 but could offer operating guidance for both Tucano and the GMC mines.

For Tucano, 2020 planned production is between 120,000 and 130,000 gold ounces - up around between 13% and 23%.

At GMC, planned production is between 1.2M to 1.4M silver equivalent ounces, a decline of ~7% to 20%

Previously: Great Panther Mining EPS misses by $0.06, beats on revenue (March 31)

Original post:
Great Panther Mining focusing on exploration, resource expansion and finances for 2020 - Seeking Alpha

Recommendation and review posted by Bethany Smith

MONROVIA, Calif.--(BUSINESS WIRE)--Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer and autoimmune disease, today announced it has entered into a technology license agreement with Vir Biotechnology, Inc., in which Vir will have non-exclusive access to Xencors Xtend Fc technology to extend the half-life of novel antibodies that Vir is investigating as potential treatments for patients with COVID-19, the disease caused by the novel coronavirus SARS-CoV-2.

The COVID-19 crisis requires urgent and coordinated action by the biotechnology industry to develop new drugs and vaccines. Xtend Fc technology has demonstrated, in multiple antibodies and through numerous human clinical trials, the ability to extend antibody drug half-life and reduce dosing frequency in patients, an important feature in anti-viral therapy for pandemic use, said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. We are committed to broadly using Xtend technology, and our other XmAb tools, to rapidly develop potential treatments for COVID-19. Virs antibody candidates, supported by their deep infectious disease expertise, are a promising approach for treating coronavirus infections.

Under the terms of the agreement, Vir will be solely responsible for the activities and costs related to research, development, regulatory and commercial activities. Financial terms of the agreement were not disclosed. Xencor and Vir previously entered into a separate technology license agreement in August 2019, in which Xencor provided a non-exclusive license to Xtend technology for Vir's use in developing and commercializing antibodies as potential treatments for patients with influenza and hepatitis B virus infection.

Xencor continues to evaluate the potential impact of the COVID-19 pandemic on ongoing and planned clinical studies. The Company is currently maintaining preestablished guidance on 2020 corporate milestones and will provide additional updates as needed.

About Xtend XmAb Fc Technology

Xencors Xtend XmAb Fc domains increase circulating half-life by increasing binding affinity to the receptor FcRn. FcRn is present inside lysosomes in endothelial cells lining the blood vessels and functions to rescue antibodies from the degradation that makes most proteins short-lived in circulation. Half-life extension can be exploited to potentially improve therapeutic antibody performance in several ways, such as increasing dosing interval or decreasing drug quantities at the same dosing interval compared to a parent antibody. Xtend technology is currently in multiple clinical-stage programs and one approved therapy, Alexions Ultomiris (ravulizumab-cwvz).

About Xencor, Inc.

Xencor is a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer and autoimmune diseases. Currently, 15 candidates engineered with Xencor's XmAb technology are in clinical development internally and with partners. Xencor's XmAb antibody engineering technology enables small changes to the structure of monoclonal antibodies resulting in new mechanisms of therapeutic action. For more information, please visit http://www.xencor.com.

Xencor Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of applicable securities laws, including, but not limited to, the quotations from Xencor's president and chief executive officer and any expectations relating to Xencor's technology, clinical trials, patient outcomes, future product candidates, research and development programs, regulatory and commercialization activities, partnering efforts and business. Such statements involve known and unknown risks, uncertainties and other factors that may cause actual results, performance or achievements and the timing of events to be materially different from those implied by such statements, and therefore these statements should not be read as guarantees of future performance or results. Such risks include, without limitation, the risks associated with the process of discovering, developing, manufacturing and commercializing drugs that are safe and effective for use as human therapeutics and other risks described in Xencor's public securities filings. For a discussion of these and other factors, please refer to Xencor's annual report on Form 10-K for the year ended December 31, 2019 as well as Xencor's subsequent filings with the Securities and Exchange Commission. All forward-looking statements are based on Xencor's current information and belief as well as assumptions made by Xencor. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are qualified in their entirety by this cautionary statement and Xencor undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law.

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Xencor and Vir Biotechnology Enter License Agreement for Use of Xtend XmAb Antibody Technology in Investigational Antibodies to Treat COVID-19 -...

Recommendation and review posted by Bethany Smith

Newswise Women undergoing radiotherapy for many cancers are more likely than men to be cured, but the side effects are more brutal, according to one of Australias most experienced radiation oncology medical physicists.

University of South Australia (UniSA) Professor of Medical Radiation, Eva Bezak, says women are generally more sensitive to radiation than men, but this is not considered in international guidelines for radiation dosages.

Current guidelines are generally based on a persons height, weight or BMI, and radiobiological responses of the general population.

In a paper published in Critical Reviews in Oncology/Hematology, Professor Bezak and her colleagues Louis de Courcy from University College Dublin and Professor Loredana Marcu from the University of Oradea in Romania highlight the need for gender to be taken into account when administering radiation.

It is clear that gender plays a role in the occurrence and response to therapy of many diseases, Professor Bezak says.

For example, it is already well established that men are more susceptible to head, neck and blood cancers and women are more prone to auto immune diseases as well as developing osteoporosis.

Scientists also know that individual responses to radiotherapy are up to 80 per cent determined by genetics.

So, where do we start with gendered medicine?

The next step is to ensure that we use both male and female mice even in our pre-clinical testing so we can get a better understanding of how gender influences treatment outcomes.

It is also important to collect data retrospectively so we can compare the radiotherapy outcomes for men and women who were prescribed radiotherapy for the same cancer.

It is a double-edged coin for men, too. Because they are more radio-resistant than women, their healthy tissues are better protected when receiving radiotherapy with fewer side effects, but their long-term survival rates are shorter.

The differences in radiation responses are highlighted by two major events in history: the Chernobyl nuclear reaction disaster in 1986 and the atomic bombings of Hiroshima and Nagasaki in 1945.

Professor Bezak says following Hiroshima and Nagasaki, the incidence of cancer in Japan was much higher in women (58 per cent) compared to men (35 per cent).

Likewise, after the Chernobyl nuclear accident, millions fewer girls were born to irradiated men and women were at greater risk of endocrine imbalance, thyroid cancer and brain tumours.

The one area that does appear to give women some protection against radiation is the female hormone oestrogen, which has a neuroprotective effect during head irradiation.

As healthcare becomes progressively more tailored to the individual, gender is a factor that can no longer be disregarded. It needs to be taken into account as an independent prognostic factor, Prof Bezak says.

A video explaining the differences in radiation outcomes between men and women can be viewed at https://youtu.be/BtDniRA7DMs

Notes for editors

Gender-dependent radiotherapy: the next step in personalised medicine? is published in Critical Reviews in Oncology/Hematology. The other contributing authors are Louis de Courcy from University College, Dublin, and Professor Loredana G. Marcu from the University of Oradea.

Visit link:
The pros and cons of radiotherapy: will it work for you? - Newswise

Recommendation and review posted by Bethany Smith

TORONTO , March 31, 2020 /CNW/ - The Gairdner Foundation is pleased to announce the 2020 Canada Gairdner Award laureates, recognizing some of the world's most significant biomedical research and discoveries. During these challenging times, we believe it is important to celebrate scientists and innovators from around the world and commend them for their tireless efforts to conduct research that impacts human health.

2020 Canada Gairdner International AwardThe five 2020 Canada Gairdner International Award laureates are recognized for seminal discoveries or contributions to biomedical science:

Dr. Masatoshi Takeichi Senior Visiting Scientist, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan ; Professor Emeritus, Kyoto University , Kyoto, Japan

Dr. Rolf Kemler Emeritus Member and Director, Max Planck Institute of Immunobiology and Epigenetics, Freiburg, Germany

Awarded "For their discovery, characterization and biology of cadherins and associated proteins in animal cell adhesion and signalling."

Dr. Takeichi

The Work: The animal body is made up of numerous cells. Dr. Takeichi was investigatinghow animal cells stick together to form tissues and organs, and identified a key protein which he named 'cadherin'.Cadherin is present on the surface of a cell and binds to the same cadherin protein on the surface of another cell through like-like interaction, thereby binding the cells together. Without cadherin, cell to cell adhesion becomes weakened and leads to the disorganization of tissues. Dr. Takeichi found that there are multiple kinds of cadherin within the body, each of which are made by different cell types, such as epithelial and neuronal cells. Cells with the same cadherins tend to cluster together, explaining the mechanism of how different cells are sorted out and organized to form functional organs.

Further studies by Dr. Takeichi's group showed that cadherin function is supported by a number of cytoplasmic proteins, includingcatenins, and their cooperation is essential for shaping of tissues. His studies also revealed that the cadherin-dependent adhesion mechanism is involved in synaptic connections between neurons, which are important for brain wiring.

Dr. Kemler

The Work: Dr. Kemler, using an immunological approach, developed antibodies directed against surface antigens of early mouse embryos. These antibodies were shown to prevent compaction of the mouse embryo and interfered with subsequent development. Both Dr. Kemler and Dr. Takeichi went on to clone and sequence the gene encoding E-cadherin and demonstrate that it was governing homophilic cell adhesion.

Dr. Kemler also discovered the other proteins that interact with the cadherins, especially the catenins, to generate the machinery involved in animal cell-to-cell adhesion. This provided the first evidence of their importance in normal development and diseases such as cancer. It has been discovered that cadherins and catenins are correlated to the formation and growth of some cancers and how tumors continue to grow. Beta catenin is linked to cell adhesion through interaction with cadherins but is also a key component of the Wnt signalling pathway that is involved in normal development and cancer. There are approximately 100 types of cadherins, known as the cadherin superfamily.

Dr. Takeichi

The Impact: The discovery of cadherins, which are found in all multicellular animalspecies, has allowed us to interpret how multicellular systems are generated and regulated. Loss of cadherin function has been implicated as the cause of certain cancers, as well as in invasiveness of many cancers. Mutations in special types of cadherin result in neurological disorders, such as epilepsy and hearing loss. The knowledge of cadherin function is expected to contribute to the development of effective treatments against such diseases.

Dr. Kemler

The Impact: Human tumors are often of epithelial origin. Given the role of E-cadherin for the integrity of an epithelial cell layer, the protein can be considered as a suppressor of tumor growth. The research on the cadherin superfamily has had great impact on fields as diverse as developmental biology, cell biology, oncology, immunology and neuroscience. Mutations in cadherins/catenins are frequently found in tumors. Various screens are being used to identify small molecules that might restore cell adhesion as a potential cancer therapy.

Dr. Roel Nusse Professor & Chair, Department of Developmental Biology; Member, Institute for StemCell Biology andRegenerativeMedicine, Stanford University , School of Medicine. Virginia and Daniel K. Ludwig Professor of Cancer Research. Investigator, Howard Hughes Medical Institute

Awarded"For pioneering work on the Wnt signaling pathway and its importance in development, cancer and stem cells"

The Work: Dr. Nusse's research has elucidated the mechanism and role of Wnt signaling, one of the most important signaling systems in development. There is now abundant evidence that Wnt signaling is active in cancer and in control of proliferation versus differentiation of adult stem cells, making the Wnt pathway one of the paradigms for the fundamental connections between normal development and cancer.

Among Dr. Nusse's contributions is the original discovery of the first Wnt gene (together with Harold Varmus) as an oncogene in mouse breast cancer. Afterwards Dr. Nusse identified the Drosophila Wnt homolog as a key developmental gene, Wingless. This led to the general realization of the remarkable links between normal development and cancer, now one of the main themes in cancer research. Using Drosophila genetics, he established the function of beta-catenin as a mediator of Wnt signaling and the Frizzleds as Wnt receptors (with Jeremy Nathans ), thereby establishing core elements of what is now called the Wnt pathway. A major later accomplishment of his group was the first successful purification of active Wnt proteins, showing that they are lipid-modified and act as stem cell growth factors.

The Impact: Wnt signaling is implicated in the growth of human embryos and the maintenance of tissues. Consequently, elucidating the Wnt pathway is leading to deeper insights into degenerative diseases and the development of new therapeutics. The widespread role of Wnt signaling in cancer is significant for the treatment of the disease as well. Isolating active Wnt proteins has led to the use of Wnts by researchers world-wide as stem cell growth factors and the expansion of stem cells into organ-like structures (organoids).

Dr. Mina J. Bissell Distinguished Senior Scientist, Biological Systems and Engineering Division, Lawrence Berkeley National Laboratory; Faculty; Graduate Groups in Comparative Biochemistry, Endocrinology, Molecular Toxicology and Bioengineering, University of California Berkeley , Berkeley, CA , USA

Awarded "For characterizing "Dynamic Reciprocity" and the significant role that extracellular matrix (ECM) signaling and microenvironment play in gene regulation in normal and malignant cells, revolutionizing the fields of oncology and tissue homeostasis."

The Work: Dr. Mina Bissell's career has been driven by challenging established paradigms in cellular and developmental biology. Through her research, Dr. Bissell showed that tissue architecture plays a dominant role in determining cell and tissue phenotype and proposed the model of 'dynamic reciprocity' (DR) between the extracellular matrix (ECM) and chromatin within the cell nucleus. Dynamic reciprocity refers to the ongoing, bidirectional interaction between cells and their microenvironment. She demonstrated that the ECM could regulate gene expression just as gene expression could regulate ECM, and that these two phenomena could occur concurrently in normal or diseased tissue.

She also developed 3D culture systems to study the interaction of the microenvironment and tissue organization and growth, using the mammary gland as a model.

The Impact:Dr. Bissell's model of dynamic reciprocity has been proven and thoroughly established since its proposal three decades ago and the implications have permeated every area of cell and cancer biology, with significant implications for current and future therapies. Dr. Bissell's work has generated a fundamental and translationally crucial paradigm shift in our understanding of both normal and malignant tissues.

Her findings have had profound implications for cancer therapy by demonstrating that tumor cells can be influenced by their environment and are not just the product of their genetic mutations. For example, cells from the mammary glands grown in two-dimensional tissue cultures rapidly lose their identity, but once placed in proper three-dimensional microenvironments, they regain mammary form and function. This work presages the current excitement about generation of 3D tissue organoids and demonstrates Dr. Bissell's creative and innovative approach to science.

Dr. Elaine Fuchs Howard Hughes Medical Institute Investigator and Rebecca C. Lancefield Professor and Head of the Robin Chemers Neustein Laboratory of Mammalian Cell Biology and Cell Biology; The Rockefeller University , New York, NY , USA

Awarded"For her studies elucidating the role of tissue stem cells in homeostasis, wound repair, inflammation and cancer."

The Work: Dr. Fuchs has used skin to study how the tissues of our body are able to replace dying cells and repair wounds. The skin must replenish itself constantly to protect against dehydration and harmful microbes. In her research, Fuchs showed that this is accomplished by a resident population of adult stem cells that continually generates a shell of indestructible cells that cover our body surface.

In her early research, Fuchs identified the proteins---keratinsthat produce the iron framework of the skin's building blocks, and showed that mutations in keratins are responsible for a group of blistering diseases in humans. In her later work, Fuchs identified the signals that prompt skin stem cells to make tissue and when to stop. In studying these processes, Fuchs learned that cancers hijack the fundamental mechanisms that tissue stem cells use to repair wounds. Her team pursued this parallel and isolated and characterized the malignant stem cells that are responsible for propagating a type of cancer called "squamous cell carcinoma." In her most recent work, she showed that these cells can be resistant to chemotherapies and immunotherapies and lead to tumor relapse.

The Impact: All tissues of our body must be able to replace dying cells and repair local wounds. Skin is particularly adept at performing these tasks. The identification and characterization of the resident skin stem cells that make and replenish the epidermis, sweat glands and hair provide important insights into this fountain of youth process and hold promise for regenerative medicine and aging. In normal tissues, the self-renewing ability of stem cells to proliferate is held in check by local inhibitory signals coming from the stem cells' neighbours. In injury, stimulatory signals mobilize the stem cells to proliferate and repair the wound. In aging, these normal balancing cues are tipped in favour of quiescence. In inflammatory disorders, stem cells become hyperactivated. In cancers, the wound mechanisms to mobilize stem cells are hijacked, leading to uncontrolled tissue growth. Understanding the basic mechanisms controlling stem cells in their native tissue is providing new strategies for searching out refractory tumor cells in cancer and for restoring normalcy in inflammatory conditions.

2020 John Dirks Canada Gairdner Global Health AwardThe 2020 John Dirks Canada Gairdner Global Health Award laureate is recognized for outstanding achievements in global health research:

Professor Salim S. Abdool Karim Director of CAPRISA (Centre for the AIDS Program of Research in South Africa), the CAPRISA Professor in Global Health at Columbia University , New York and Pro Vice-Chancellor (Research) at the University of KwaZulu-Natal, Durban, South Africa

Professor Quarraisha Abdool KarimAssociate Scientific Director of CAPRISA, Professor in Clinical Epidemiology, Columbia University , New York and Professor in Public Health at the Nelson Mandela Medical School and Pro Vice-Chancellor (African Health) at the University of KwaZulu-Natal, Durban, South Africa

Awarded"For their discovery that antiretrovirals prevent sexual transmission of HIV, which laid the foundations for pre-exposure prophylaxis (PrEP), the HIV prevention strategy that is contributing to the reduction of HIV infection in Africa and around the world."

The Work: UNAIDS estimates that 37 million people were living with HIV and 1.8 million people acquired HIV in 2017. In Africa, which has over two thirds of all people with HIV, adolescent girls and young women have the highest rates of new HIV infections. ABC (Abstinence, Be faithful, and use Condoms) prevention messages have had little impact - due to gender power imbalances, young women are often unable to successfully negotiate condom use, insist on mutual monogamy, or convince their male partners to have an HIV test.

In responding to this crisis, Salim and Quarraisha Abdool Karim started investigating new HIV prevention technologies for women about 30 years ago. After two unsuccessful decades, their perseverance paid off when they provided proof-of-concept that antiretrovirals prevent sexually acquired HIV infection in women. Their ground-breaking CAPRISA 004 trial showed that tenofovir gel prevents both HIV infection and genital herpes. The finding was ranked inthe "Top 10 Scientific Breakthroughs of 2010" by the journal, Science. The finding was heralded by UNAIDS and the World Health Organization (WHO) as one of the most significant scientific breakthroughs in AIDS and provided the first evidence for what is today known as HIV pre-exposure prophylaxis (PrEP).

The Abdool Karims have also elucidated the evolving nature of the HIV epidemic in Africa , characterising the key social, behavioural and biological risk factors responsible for the disproportionately high HIV burden in young women. Their identification of the "Cycle of HIV Transmission", where teenage girls acquire HIV from men about 10 years older on average, has shaped UNAIDS policies on HIV prevention in Africa .

The impact: CAPRISA 004 and several clinical trials of oral tenofovir led tothe WHO recommending a daily tenofovir-containing pill for PrEP as a standard HIV prevention tool for all those at high risk a few years later. Several African countries are among the 68 countries across all continents that are currently making PrEP available for HIV prevention. The research undertaken in Africa by this South African couple has played a key role in shaping the local and global response to the HIV epidemic.

2020 Canada Gairdner Wightman AwardThe 2020 Canada Gairdner Wightman Award laureate is a Canadian scientist recognized for outstanding leadership in medicine and medical science throughout their career:

Dr. Guy Rouleau Director of the Montreal Neurological Institute-Hospital (The Neuro); Professor & Chair of the Department of Neurology and Neurosurgery, McGill University ; Director of the Department of Neuroscience, McGill University Health Center

Awarded "For identifying and elucidating the genetic architecture of neurological and psychiatric diseases, including ALS, autism and schizophrenia, and his leadership in the field of Open Science."

The Work: Dr. Rouleau has identified over 20 genetic risk factors predisposing to a range of brain disorders, both neurological and psychiatric, involving either neurodevelopmental processes or degenerative events. He has defined a novel disease mechanism for diseases related to repeat expansions that are at play in some of the most severe neurodegenerative conditions. He has significantly contributed to the understanding of the role of de novo variants in autism and schizophrenia. In addition, he has made important advances for various neuropathies, in particular for amyotrophic lateral sclerosis (ALS) where he was involved in the identification of the most prevalent genetic risk factors -which in turn are now the core of innumerable ALS studies worldwide.

Dr. Rouleau has also played a pioneering role in the practice of Open Science (OS), transforming the Montreal Neurological Institute-Hospital (The Neuro) into the first OS institution in the world. The Neuro now uses OS principles to transform research and careand accelerate the development of new treatments for patients through Open Access, Open Data, Open Biobanking, Open Early Drug Discovery and non-restrictive intellectual property.

The Impact: The identification of genetic risk factors has a number of significant consequences. First, allowing for more accurate genetic counselling, which reduces the burden of disease to affected individuals, parents and society. A revealing case is Andermann syndrome, a severe neurodevelopmental and neurodegenerative condition that was once relatively common in the Saguenay-Lac-St-Jean region of Quebec . Now this disease has almost disappeared from that population. Second, identifying the causative gene allows the development of treatments. For instance, his earlier work on a form of ALS linked to the superoxide dismutase-1 gene (SOD1) opened up studies which are now the focal point of phase 2 clinical studies showing great promise.

Byactingasalivinglabforthelast coupleofyears,TheNeuroisspearheading the practice of OpenScience (OS).TheNeurois alsoengagingstakeholdersacross Canadawiththegoal of formalizinganational OSallianceforthe neurosciences.Dr.Rouleau'sworkinOScontributesfundamentallytothetransformationoftheveryecosystemofsciencebystimulatingnewthinkingandfosteringcommunitiesofsharing.InspiredbyTheNeuro'svision,theglobalsciencecommunityisreflecting oncurrentresearchconventionsandcollaborativeprojects,andthemomentumforOSisgainingafootholdinorganizationsandinstitutionsinallcornersoftheearth.

About the Gairdner Foundation:

The Gairdner Foundation was established in 1957 by Toronto stockbroker, James Gairdner to award annual prizes to scientists whose discoveries have had major impact on scientific progress and on human health. Since 1959 when the first awards were granted, 387scientists have received a Canada Gairdner Award and 92 to date have gone on to receive the Nobel Prize.The Canada Gairdner Awards promote a stronger culture of research and innovation across the country through our Outreach Programs including lectures and research symposia. The programs bring current and past laureates to a minimum of 15 universities across Canada to speak with faculty, trainees and high school students to inspire the next generation of researchers. Annual research symposia and public lectures are organized across Canada to provide Canadians access to leading science through Gairdner's convening power.

http://www.gairdner.org

SOURCE Gairdner Foundation

View original content to download multimedia: http://www.newswire.ca/en/releases/archive/March2020/31/c7291.html

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2020 Canada Gairdner Awards Recognize World-renowned Scientists for Transformative Contributions to Research That Impact Human Health - Yahoo Finance

Recommendation and review posted by Bethany Smith

Mar. 27 /CSRwire/ - As an international non-profit organization, every day DKMS provides 20 lifesaving blood stem cell transplants for blood cancer and blood disorder patients all around the world. Even in the current situation that we are facing with COVID-19, these patients still urgently need our help. It is for this reason that we keep fighting and continue to work together and collaborate with the international blood stem cell community to be able to overcome all barriers, to give as many patients as possible, who are in urgent need of a life-saving blood stem cell transplant, with a second chance at life.

IMPORTANCE OF REGISTRATION

Due to the advice and directions from the health authorities regarding the COVID-19 virus and to play our part to flatten the curve, we have postponed all of our planned donor drives/registration events in all of our six countries. We want to make sure that we look after and protect all of our donors and volunteers. For the thousands of patients around the world who are not able to find a perfect match, we still urgently need to add more donors to the database. We are thankful for every registration and you can still order your registration kit online atwww.dkms.org. Registering is simple and straightforward and you can become a potential lifesaver!

PROTECTING OUR DONORS

Patients around the world still urgently need blood stem cell transplants to survive. This is why we ask all of our donors to make themselves available if they are a match for a patient. The health and well-being of our donors is our number one priority and we consider every case on an individual basis. We will always do everything possible to ensure the safety of our donors.

We have implemented screening for the COVID-19 virus risks before entry to collection centers and before the start of a donation.

These measures serve to protect the donor as well as the recipient and the employees in the collection centers. Health experts have confirmed that there is currently no evidence that the COVID-19 virus can be transmitted via blood, bone marrow, or stem cell products.

We are grateful for every one of our incredible donors and for their commitment to providing patients with a second chance at life.

TRANSPORT OF BLOOD STEM CELL PRODUCTS

75% of all of our life-saving blood stem cell collections from DKMS donors travel across borders to patients in other countries. Due to the travel restrictions imposed by many countries because of the COVID-19 virus, we are faced with additional challenges in ensuring patients receive their transplant.

When it comes to transplants, time is crucial and we are determined to find solutions to be able to provide patients in need with their transplant. We are joining forces with other members of the international transplant community and national and international authorities in order to overcome these challenges. Despite the added difficulties that we are facing at the moment, as always, we are determined to overcome all barriers in order to provide as many patients as possible with a second chance at life.

FAQ's

1. WHY HAVE DONOR DRIVES BEEN CANCELLED OR POSTPONED AND WHAT IS THE ALTERNATIVE?

In light of the increasing spread of the COVID-19 virus, DKMS is postponing all planned events and registration drives for new potential blood stem cell donors until further notice. DKMS sees itself with a special duty of care and with these measures specifically follows the recommendations of the Centers for Disease Control to protect of the population.

Blood cancer patients around the world are still in urgent need of bone marrow and blood stem cell transplants and many are still not able to find a matching donor. This is why DKMS continues to encourage everyone that is able to, to register as a blood stem cell donor and if required, to also make themselves available for a donation."We must not abandon any patient who is currently searching for a blood stem cell donor or waiting for a life-saving blood stem cell transplant. In this difficult situation, I urgently ask that everyone continues to make themselves available as donors. We are taking all precautions necessary to protect each and every one of our donors in the best possible way," said Dr. Elke Neujahr, Global CEO DKMS.

We are grateful for every registration and to enable everyone that is interested to be able to register, DKMS offers the possibility of virtual donor drives that gives initiators, families and friends the opportunity to host a virtual drive to add more potential lifesavers to the donor pool via our online platform which can be found by clicking herehttps://dkmsgetinvolved.org/virtual-donor-drive. With this capability, any planned on ground registration drives can take place digitally instead and potential donors, which are urgently needed, can still register. The links to the online registration drives can be shared via all channels.

As always, registering as a blood stem cell donor via our homepage atwww.dkms.orgis also possible. Anyone who is interested can easily order a registration kit to be sent to their home. With the help of three cotton swabs and clear instructions, as well as a consent form, every potential new donor can take a cheek swab themselves and send it via post back to DKMS. In the DKMS Life Science Lab the individual HLA characteristics (also: tissue characteristics) of the donor are then analyzed, allowing the potential donor to be registered in the DKMS database.

The difference between the online registration drives and the general online registration is that the online registration drives are linked to particular patient appeals and the number of donors can be tracked accordingly. However, in general, it doesnt matter how you register, you will always be available as a potential lifesaver for blood cancer and blood disorder patients searching for a matching donor all over the world!

2. AM I STILL ABLE TO REGISTER ONLINE IF I SUSPECT THAT I MAY HAVE OR DO HAVE COVID- 19?

If it has been confirmed that you have been infected with the COVID-19 virus or you are awaiting test results, you unfortunately will not be able to register until 3 months after the infection has passed.

3. I REGISTERED ONLINE AND HAVENT RECEIVED A CONFIRMATION, IS THERE A DELAY?

We ask anyone registering as a potential donor online for their understanding. Normally we send all registration kits that we receive to our lab within 24 hours, however we are currently waiting 7 days until we do this to ensure the protection of our staff handling the returned registration kits. Please be assured that the analysis of your sample and HLA characteristics that is required for us to add you to the register, will take place as soon as possible.

4. HOW WILL DKMS MAINTAIN THE SAFETY OF DONORS?

The health and safety of our donors is our highest priority. We will consider the circumstances of all of our donors who are asked to donate their bone marrow and blood stem cells, on an individual basis, especially regarding their travel to a collection center. Please be reassured that we provide the best and safest solution possible for our donors.

Unfortunately, donors who live in high risk areas have to be blocked for search requests, as they are currently not eligible to proceed with a donation. We also have implemented screening for the COVID-19 virus risks before entry to any collection center and before the start of a donation. These measures serve to protect the donor as well as the recipient and the employees in the collection centers. Health experts have confirmed that there is currently no evidence that the novel Coronavirus can be transmitted via blood, bone marrow, or stem cell products.

Please also understand that any planned collection may be postponed or cancelled due to the current situation. We will keep you informed about all developments. Regarding the transport of blood stem cell products to the respective transplant clinics, we are in regular and close contact with all parties involved as well as with all relevant national and international authorities and organizations, to ensure that all blood stem cell collections can reach the patients.

5. IS THERE AN INCREASED CHANCE OF DONORS CONTRACTING THE COVID-19 VIRUS DURING THE DONATION PROCEDURE OR PREPARATION FOR THE PR

There is no risk of contracting the COVID-19 virus as a result of a blood stem cell collection. The health and safety of our donors is our number one priority and we will always ensure all procedures are in place to protect their safety.

6. CAN DONORS POSTPONE THEIR DONATION AT THIS TIME?

The final decision to donate is always made by the actual donor. If you have been asked to donate and have any concerns, please contact your DKMS coordinator. We evaluate each case on an individual basis to find the best solution for all parties involved.

7. IS IT STILL SAFE FOR PATIENTS TO RECEIVE A TRANSPLANT?

Health experts have confirmed that there is currently no evidence that the novel Coronavirus can be transmitted via blood, bone marrow, or stem cell products.

8. HOW IS THE TRANSPORT OF COLLECTIONS FROM DONORS IMPACTED BY THE CURRENT TRAVEL RESTRICTIONS?

The majority of blood stem cell collections from DKMS donors travel across borders to patients in other countries. Due to the travel restrictions imposed by many countries we are in regular and close contact with all parties involved in the transport, as well as with the relevant national and international authorities and organizations. Despite the added difficulties that we are facing at the moment, we are, as always, determined to overcome all barriers in order to provide as many patients as possible with a second chance at life.

9. HOW IS DKMS RESPONDING TO THE CURRENT TRAVEL RESTRICTIONS IN THE US?

Due to the restrictions on entry into the USA, DKMS has been in contact with the World Marrow Donor Association (WMDA) and the National Marrow Donor Program (NMDP). Together, we obtained special permits for stem cell couriers from Germany and Europe to enter the USA.

10. HOW WILL THE SPREAD OF THE COVID-19 VIRUS AFFECT DKMS AND ITS OPERATIONS?

As with every organization across the world, DKMS faces challenges caused by COVID-19 and some of the potential impacts are still not yet known. The goal of DKMS remains the same as it always has been and that is, together with our outstanding team of donors and employees, to give as many patients as possible around the world a second chance at life, through a life-saving bone marrow and blood stem cell transplant. The management team of DKMS is working around the clock together with all DKMS employees and relevant stakeholders to achieve this goal.

11. HOW WILL DKMS RESPOND AS THE SITUATION EVOLVES?

A dedicated team made up of experts from relevant departments at DKMS is constantly monitoring and evaluating the ongoing situation and will act accordingly. We will continue to keep our donors and all stakeholders updated.

12. IS DKMS TESTING POTENTIAL DONORS DURING THE REGISTRATION PROCESS?

DKMS does not test potential donors who register with us for the COVID-19 virus. If you are confirmed to have the virus, we ask that you do not register with us for the next 3 months. For donors who are asked to donate we have implemented screening for the COVID-19 virus risks before entering any collection center and before the start of a donation. These measures serve to protect the donor as well as the recipient and the employees in the collection center.

13. IF I AM A POTENTIAL DONOR AND I HAVE QUESTIONS, WHO SHOULD I CONTACT?

If you have been contacted by DKMS as a potential donor and if you have any questions or concerns, you are encourage to contact your DKMS coordinator. As always, DKMS staff is there to help you through the process and answer any questions that you may have.

14. IS THERE A NEED FOR QUARANTINE OR TESTING OF STEM CELL PRODUCTS BEFORE TRANSPLANTATION?

Although SARS-CoV and MERS-CoV have been detected in blood, there have not been any reports of transmission from donor to recipient either in transfusion of blood products or cellular therapies. As there is no evidence of transmission via blood, bone marrow, or stem cell products DKMS endorses the recommendations of the regulatory agencies such as the FDA and AABB which currently do not recommend or require product testing or to quarantine the stem cell product. For more information, please visit:share.wmda.info.

If a patient is in need of a transplant, there is no need to wait or prolong the process by testing or quarantining the stem cell product. Planned transplantations can go ahead.

See the article here:
Now More Than Ever, the Importance of Second Chances at Life - CSRwire.com

Recommendation and review posted by Bethany Smith

VANCOUVER, Washington, March 30, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today that an additional three critically ill COVID-19 patients have been treated with leronlimab. These additional patients increase the total to 10 patients receiving leronlimab treatment under an Emergency Investigational New Drug (EIND) granted by the U.S. Food and Drug Administration (FDA).

The treatment with leronlimab is targeted as a therapy for patients who experience respiratory complications as a result of contracting SARS-CoV-2 causing the Coronavirus Disease 2019 (COVID-19). Leronlimab is believed to provide therapeutic benefit by enhancing the immune response while mitigating the cytokine storm that leads to morbidity and mortality in these patients. The laboratory evaluation of the first four patients treated with leronlimab revealed that the immune profile in these patients approached normal levels and the levels of cytokines involved in the cytokine storm (including IL-6 and TNF alpha) were much improved. The results of the three additional patients are expected this week.

Jacob Lalezari, M.D., Interim Chief Medical Officer of CytoDyn, commented, The preliminary results observed in patients who were severely ill with COVID-19 and treated with leronlimab are encouraging. Although the data set is still small, we saw fairly rapid and positive laboratory responses in all 4 patients treated, and in three of the 4 patients these laboratory results were associated with a favorable clinical outcome. We eagerly await the results of additional patients treated under the FDAs emergency IND program, as well as the results of several randomized clinical trials about to start.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, added, We remain encouraged and hopeful that leronlimab will help patients from this devastating and relentless disease. We will aggressively pursue treatment for COVID-19 patients, and to explore leronlimabs role in helping to alleviate the impending burden of supply chain and institutional capacity issues.

About Coronavirus Disease 2019SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140) The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH. Leronlimab has completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDyn CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in April of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking Statements This press release contains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Companys cash position, (ii) the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv) the Companys ability to enter into partnership or licensing arrangements with third parties, (v) the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Companys ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Companys clinical trials, (viii) the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors: Dave Gentry, CEO RedChip Companies Office: 1.800.RED.CHIP (733.2447) Cell: 407.491.4498 dave@redchip.com

Read the rest here:
Three Additional Patients with Severe COVID-19 Treated with Leronlimab in New York Medical Center Bringing the Total to 10 Patients - Associated Press

Recommendation and review posted by Bethany Smith